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1. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

Author

Strauss, Kevin A., Farrar, Michelle A., Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R., Servais, Laurent, McMillan, Hugh J., Finkel, Richard S., Swoboda, Kathryn J., Kwon, Jennifer M., Zaidman, Craig M., Chiriboga, Claudia A., Iannaccone, Susan T., Krueger, Jena M., Parsons, Julie A., Shieh, Perry B., Kavanagh, Sarah, Tauscher-Wisniewski, Sitra, McGill, Bryan E., and Macek, Thomas A.

Published
2022
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2. Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

Author

Strauss, Kevin A., Farrar, Michelle A., Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R., Servais, Laurent, McMillan, Hugh J., Finkel, Richard S., Swoboda, Kathryn J., Kwon, Jennifer M., Zaidman, Craig M., Chiriboga, Claudia A., Iannaccone, Susan T., Krueger, Jena M., Parsons, Julie A., Shieh, Perry B., Kavanagh, Sarah, Wigderson, Melissa, Tauscher-Wisniewski, Sitra, McGill, Bryan E., and Macek, Thomas A.

Published
2022
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7. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

Author

Day, John W, Finkel, Richard S, Chiriboga, Claudia A, Connolly, Anne M, Crawford, Thomas O, Darras, Basil T, Iannaccone, Susan T, Kuntz, Nancy L, Peña, Loren D M, Shieh, Perry B, Smith, Edward C, Kwon, Jennifer M, Zaidman, Craig M, Schultz, Meredith, Feltner, Douglas E, Tauscher-Wisniewski, Sitra, Ouyang, Haojun, Chand, Deepa H, Sproule, Douglas M, Macek, Thomas A, and Mendell, Jerry R

Published
2021
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25. Contributors

Author

Abzug, Mark J., Acharya, Krishna K., Adams, Denise M., Adelson, Stewart, Adrian, Molly C., Ahlfeld, Shawn K., Aiken, John J., Akdis, Cezmi A., Albokhari, Daniah, Alderman, Elizabeth M., Ali, Omar, Allen-Rhoades, Wendy A., Almutlaq, Nourah N., Amos, Louella B., Anari, Jason B., Anderson, Karl E., Anupindi, Sudha A., Appleby, Brian S., Ardoin, Stacy P., Arkader, Alexandre, Armangué, Thaís, Arndt, Carola A.S., Arnold, Danielle E., Artis, Adrianne R., Asher, David M., Asselin, Barbara L., Astley, Christina M., Atkinson, Norrell K., Augustine, Erika F., Augustyn, Marilyn C., Bacharier, Leonard B., Bacino, Carlos A., Bailey, Zinzi D., Balamuth, Frances B., Baldassano, Robert N., Baldwin, Keith D., Bales, Christina B., Balistreri, William F., Balwani, Manisha, Bamba, Vaneeta, Banerji, Aleena, Bang, Janet Y., Barai, Nikita, Baranowski, Katherine, Barclay, Sarah F., Barkoudah, Elizabeth, Barrero-Castillero, Alejandra, Barrett, Katherine J., Barron, Karyl S., Basel, Donald, Bass, Dorsey M., Bassett, Mary T., Bassiri, Hamid, Baum, Rebecca A., Behrens, Edward M., Bell, Michael J., Benjamin, Daniel K., Jr., Bennett, Amanda E., Bergerson, Jenna R.E., Bernstein, Daniel, Bernstein, Henry H., Bice-Urbach, Brittany J., Bielory, Brett P., Bielory, Leonard, Blanchard, Samra S., Blanchette, Eliza, Blatter, Joshua A., Bleyer, Archie, Boas, Steven R., Bock, Margret E., Boggs, Sarah R., Boivin, Michael J., Bonn, Julie, Bonthius, Daniel J., Boppana, Suresh B., Bordini, Brett J., Borst, Alexandra J., Bosse, Kristopher R., Boyer, Kenneth M., Brady, Patrick W., Brady, Rebecca C., Brady, Samuel L., Branchford, Brian R., Brandow, Amanda M., Brandsma, Erik, Breault, David T., Breuner, Cora Collette, Bridgemohan, Carolyn F., Britt, William J., Brower, Laura, Brown, Maria D., Brownell, Jefferson N., Browning, Meghen B., Brunetti-Pierri, Nicola, Bunyavanich, Supinda, Burstein, Danielle S., Bustinduy, Amaya L., Buyon, Jill P., Cabada, Miguel M., Cada, Michaela, Cairo, Mitchell S., Calello, Diane P., Cameron, Lindsay H., Campbell, Angela J.P., Candelaria, Margo, Cannon, Laura, Carlin, Rebecca F., Carlucci, James G., Carr, Michael R., Carrigan, Robert B., Carter, Rebecca G., Carter-Hamilton, Gail V., Case, Abigail, Chang, Pearl W., Chelimsky, Gisela G., Chelimsky, Thomas, Chemaitilly, Wassim, Chiotos, Kathleen, Chiu, Yvonne E., Chong, Hey Jin, Chou, Stella T., Christ, Lori A., Christenson, John C., Chugh, Ankur A., Cieslak, Theodore J., Claes, Donna J., Coates, Thomas D., Sánchez Códez, María I., Coffin, Susan E., Cohen, Mitchell B., Cohen, Susan S., Cole, F. Sessions, III, Collaco, J. Michael, Collins, James W., Jr., Congeni, Joseph A., Conrad, Máire A., Corcoran, Justin N., Corley, Alexandra M.S., Cox, Amanda L., Coyle, Anne M., Coyne-Beasley, Tamera, Craig, Sansanee S., Creighton, Sarah M., Crigger, Chad B., Crowe, James E., Jr., Culbert, Gabriel, Czinn, Steven J., Dalal, Aarti S., Dalmau, Josep, D’Andrea, Lynn A., Danziger-Isakov, Lara A., Darville, Toni, David, Richard J., Davidoff, Katharine, Davidson, Loren T., Davidson, Richard S., Davies, H. Dele, Davis, Stephanie D., Davis-Kankanamge, Christina, Daw, Najat C., Dean, Shannon L., DeBiasi, Roberta L., Delair, Shirley, DeLaroche, Amy M., De León-Crutchlow, Diva D., Oquendo Del Toro, Helen M., Del Valle Mojica, Coralee, DeMaso, David R., Dendrinos, Melina L., Dent, Arlene E., Desnick, Robert J., Deterding, Robin R., Devarajan, Prasad, deVeber, Gabrielle A., Dhar, Vineet K., Dhossche, Julie M., Diab, Liliane K., Di Carlo, Heather N., Dietz, Harry C., III, Dietze-Fiedler, Megan L., DiMeglio, Linda A., Dixon, Bradley P., DiVasta, Amy D., Dlamini, Nomazulu, Dobbs, Katherine R., Dodhia, Sonam N., Doerholt, Katja, Dolin, Cara D., Dominguez, Samuel R., Donohoue, Patricia A., Dow, Jennifer, Downes, Kevin J., Doyle, Daniel A., Doyle, Jefferson J., Dror, Yigal, Dubowitz, Howard, Dumler, J. Stephen, Duncan, Andrea F., Durant, Nefertiti H., Dvergsten, Jeffrey A., Earing, Michael G., Eberly, Col. Matthew D., Egan, Marie E., Eichenwald, Eric C., Elkadri, Abdul-Aziz K., Englander, Elizabeth, Ericson, Jessica E., Erkan, Elif, Etzel, Ruth A., Evans, Sarah Helen, Faherty, Erin, Falk, Marni J., Familiar-Lopez, Itziar, Fargo, John H., Feemster, Kristen A., Fehnel, Katie P., Feigelman, Susan, Feldman, Amy G., Feldman, Heidi M., Fels, Edward C., Felner, Eric I., Feng, Sing-Yi, Ferkol, Thomas W., Jr., Finberg, Karin E., Finder, Jonathan D., Fiorino, Kristin N., Fischer, Philip R., Fitzpatrick, Anne M., Flannery, Dustin D., Fleming, Nicholas L., Flood, Veronica H., Flores, Francisco X., Flynn, Joseph T., Flynn, Patricia M., Foglia, Elizabeth E., Forkey, Heather C., Forman, Joel A., Freeman, Alexandra F., Friedman, Deborah M., Friedman, Susan A., Friehling, Erika D., Fritz, Stephanie A., Frush, Donald P., Fuleihan, Ramsay L., Gahagan, Sheila, Gallagher, Patrick G., Galloway, David P., Gans, Hayley A., Garber, Andrea K., Gardiner, Paula M., Garibaldi, Luigi R., Gauthier, Gregory M., Gerber, Jeffrey S., Gershon, Anne A., Ghadersohi, Saied, Gibbs, Kathleen A., Gibson, Mark, Gigante, Joseph, Gigliotti, Francis, Gilley, Stephanie P., Gilliam, Walter S., Ginde, Salil, Girotto, John A., Goldfarb, Samuel B., Goldman, David L., Goldman, Stanton C., Gómez-Duarte, Oscar G., Good, Misty, Goodbody, Christine M., Goodman, Denise M., Goodman, Tracey, Goodyer, William R., Gordon, Catherine M., Gordon, Leslie B., Gordon, Rebecca J., Gordon-Lipkin, Eliza, Gorelik, Michael, Gower, W. Adam, Graber, Evan G., Graff, Zachary T., Graham, Robert J., Green, Cori M., Green, Michael, Greenbaum, Larry A., Greenbaum, V. Jordan, Greiner, Mary V., Griffiths, Anne G., Grizzle, Kenneth L., Groner, Judith A., Grumach, Anete Sevciovic, Gueye-Ndiaye, Seyni, Guz-Mark, Anat, Haamid, Fareeda, Haddad, Gabriel G., Haddad, Joseph, Jr., Haemer, Matthew A., Hagan, Joseph F., Jr., Haider, Suraiya K., Hakim, Hana, Haldeman-Englert, Chad R., Halstead, Scott B., Hamie, Lamiaa, Hammerschlag, Margaret R., Hammershaimb, E. Adrianne, Hampton, Elisa, Hamvas, Aaron, Hanchard, Neil A., Hanley, Patrick C., Hanna, Melisha G., Harijan, Pooja D., Harrison, Douglas J., Harstad, Elizabeth B., Haslam, David B., Hauck, Fern R., Havers, Fiona P., Hayes, Ericka V., Heard-Garris, Nia J., Hedrick, Holly L., Hemingway, Cheryl, Heneghan, Chelsea, Hernandez, Michelle L., Hernandez-Trujillo, Vivian P., Hernandez Tejada, Fiorela N., Herrick, Heidi M., Hershey, Andrew D., Herzog, Cynthia E., Heston, Sarah M., Hijazi, Ghada, Hill, Samantha V., Hochberg, Jessica, Hodes, Deborah, Hoefgen, Holly R., Holinger, Lauren D., Holland-Hall, Cynthia M., Hollenbach, Laura L., Holler-Managan, Yolanda F., Hooper, David K., Hooven, Thomas A., Hoover-Fong, Julie E., Hopper, Rachel K., Hord, Jeffrey D., Horn, B. David, Horstmann, Helen M., Hotez, Peter J., House, Samantha A., Howard, Ashley C., Howard, Mary Beth, Hsu, Evelyn K., Hsu, Katherine, Huddleston, Heather G., Huh, Winston W., Humphrey, Stephen R., Hunstad, David A., Hunger, Stephen P., Hunt, Carl E., Huppert, Stacey S., Huppler, Anna R., Hurt, Hallam, Izumi, Kosuke, Jackson, Allison M., Jackson, Mary Anne, Jaffe, Ashlee M., James, Kiera M., Janowski, Andrew B., Jenssen, Brian P., Jinnah, H.A., John, Chandy C., Johansen, Kari, Johnson, Susan L., Johnston, Brian D., Jongco, Artemio M., III, Josephson, Cassandra D., Joyce, Joel C., Jyonouchi, Soma, Kabbany, Mohammad Nasser, Kabbouche, Marielle, Kacperski, Joanne, Kadry, Nadia A., Kaj-Carbaidwala, Batul, Kalish, Jennifer M., Kamat, Deepak, Kansra, Alvina R., Kanter, David M., Kao, Carol M., Kapavarapu, Prasanna K., Kattan, Jacob, Kelly, Andrea, Kelly, Desmond P., Kelly, Matthew S., Kelly, Michael E., Kendi, Sadiqa, Kerem, Eitan, Kerr, Julie M., Khan, David A., Khan, Seema, Khatami, Ameneh, Khaytin, Ilya, Kier, Catherine, Kilinsky, Alexandra, Kim, Chong-Tae, Kim, Jung Won, Kim, Rosa K., King, J. Michael, Kirschen, Matthew P., Kishnani, Priya S., Klawonn, Meghan A., Klein, Bruce L., Klein, Bruce S., Kliegman, Alison S., Kliegman, Robert M., Kneyber, Martin C.J., Koch, William C., Kochanek, Patrick M., Kodish, Eric, Kohlhoff, Stephan A., Kortepeter, Mark G., Kotloff, Karen L., Koumbourlis, Anastassios C., Krause, Peter J., Krebs, Nancy F., Kreipe, Richard E., Krug, Steven E., Kwiatkowski, Janet L., Kwon, Jennifer M., Ladisch, Stephan, Lakser, Oren J., Lalor, Leah, Lam, Simon, Lambert, Michele P., Lampe, Christina, Landry, Gregory L., Lane, Wendy G., Larson, A. Noelle, LaRussa, Phillip S., Lawrence, J. Todd R., Lee, Brendan, Lee, Erica H., Leiding, Jennifer W., Lemmon, Monica E., Lesser, Daniel J., Lestrud, Steven O., Leung, Donald Y.M., Levas, Michael N., Liacouras, Chris A., Lipkin, Paul H., Liptzin, Deborah R., Liu, Andrew H., Lo, Mindy S., Lo, Stanley F., Long, Sarah S., Lord, Katherine, Macias, Charles G., Macias, Michelle M., Macumber, Ian R., Magnusson, Mark R., Magoulas, Pilar L., Maguire, Kathleen J., Mahajan, Prashant V., Majzoub, Joseph A., Mamula, Petar, Manak, Colleen K., Mangus, Courtney W., Manoli, Irini, Manzur, Adnan Y., Maqbool, Asim, Maranich, Col. Ashley M., Margetts, Miranda, Margolis, David, Marin, Mona, Marini, Joan C., Markowitz, Morri, Maroushek, Stacene R., Marsh, Justin D., Marshall, Trisha L., Martin, Kari L., Masson, Vicki K., Matalon, Dena R., Matalon, Reuben K., Mathijssen, Irene M.J., Reddy Matta, Sravan Kumar, Maxwell, Elizabeth C., Maybank, Aletha, McCabe, Megan E., McCain, Darla H., McColley, Susanna A., McConnico, Neena, McCormick, Elizabeth M., McDonald, Christine M., McGovern, Margaret M., McGrath-Morrow, Sharon A., McInerney, Alissa, McKinney, Jeffrey S., McLeod, Rima, McVay-Gillam, Marcene R., Meade, Julia C., Meehan, William P., III, Mejias, Asuncion, Melby, Peter C., Melzer-Lange, Marlene D., Merves, Jamie F., Messacar, Kevin B., Michaels, Marian G., Michniacki, Thomas F., Mikati, Mohamad A., Miller-Handley, Hilary E., Mink, Jonathan W., Mirasola, Karolyn, Mistovich, R. Justin, Mohr, Emma L., Montoya-Williams, Diana, Moon, Rachel Y., Morava, Eva, Moreno, Megan A., Morgan, Ryan W., Morrison, Peter E., Morrison, Wynne, Mukhopadhyay, Sagori, Munoz, Flor M., Munson, David A., Murphy, Timothy F., Murray, Karen F., Murray, Thomas S., Mutlu, Levent, Nagata, Jason M., Narula, Sona, Nataro, James P., Navsaria, Dipesh, Nduati, Ruth W., Nehus, Edward J., Nelson, Maureen R., Neri, Caitlin M., Nevin, Mary A., Newburger, Jane W., Newmark, Jonathan, Nield, Linda S., Niermeyer, Susan, Nocton, James J., Nogee, Lawrence M., Noje, Corina, Nowak-Wegrzyn, Anna H., Obaro, Stephen K., Obeid, Makram M., O’Callaghan, Kevin P., Oleszek, Joyce L., Olitsky, Scott E., Olsson, John M., O’Neill, Meghan E., Onigbanjo, Mutiat T., Opoka, Robert O., Orenstein, Walter A., Orkin, Sarah H., Orscheln, Rachel C., Ortega, Camile, O’Toole, Timothy R., Owens, Judith A., Ozen, Seza, Pach, Sophie, Pachter, Lee M., Padhye, Amruta, Pandurangi, Sindhu, Pak-Gorstein, Suzinne, Palla, John, Palmieri, Tina L., Palmieri, Jessica M., Pappas, Diane E., Parent, John J., Parga-Belinkie, Joanna J., Parikh, Bijal A., Parker, Alasdair P.J., Partridge, Emily A., Patel, Ami B., Patel, Trusha, Patrick, Stephen W., Patterson, Briana C., Pelosi, Emanuele, Permar, Sallie R., Perry, Michael, Perry, Tamara T., Peters, Mark J., Peters, Timothy R., Peterson, Stacy J.B., Phelan, Rachel A., Pinto, Anna L., Pipan, Mary, Player, Brittany, Prince, William Benjamin, Proctor, Mark R., Prozora, Stephanie, Pryor, Howard I., II, Pyles, Lee A., Quinn, Molly M., Quint, Elisabeth H., Rabinovich, C. Egla, Raffini, Leslie J., Ragoonanan, Dristhi S., Rahman, Shamima, Ralston, Shawn L., Ram, Sanjay, Ramilo, Octavio, Ramirez, Kacy A., Rand, Casey M., Rasmussen, Sonja A., Rathke, Kevin M., Ratner, Adam J., Ratner, Lee, Reed, Ann M., Reich, Patrick J., Reif, Shimon, Reller, Megan E., Remick, Katherine E., Remiker, Allison S., Reyes, Jorge D., Richardson, Katherine M., Rintoul, Natalie E., Ritchey, A. Kim, Robinson, Angela Byun, Rodrigues, Kristine Knuti, Rogers, Michael E., Romano, Mary E., Roosevelt, Genie E., Roper, Stephen M., Rosenthal, Stephen M., Ross, A. Catharine, Rossano, Joseph W., Rothman, Jennifer A., Rotta, Alexandre T., Rozenfeld, Ranna A., Russo, Michael E., Ryan, Kelsey S., Ryan, Monique M., Ryu, Julie, Sabbagh, Sara E., Sachdev, H.P.S., Sadarangani, Manish, Sadun, Rebecca E., Sahin, Mustafa, Saint-Cyr, Martine, Salata, Robert A., Salazar, José H., Salvana, Edsel Maurice T., Samelson-Jones, Benjamin J., Sammons, Julia S., Sampson, Hugh A., Samsel, Chase B., Sandora, Thomas J., Sankar, Wudbhav N., Sarnaik, Ashok P., Sato, Alice I., Satter, Lisa Forbes, Scaggs Huang, Felicia A., Schaffzin, Joshua K., Schechter, Michael S., Schilling, Samantha, Schleiss, Mark R., Schluter, W. William, Schondelmeyer, Amanda C., Schroeder, James W., Jr., Schulte, Elaine E., Schuster, Jennifer E., Schuster, Marcy, Schuster, Mark A., Scott, Daryl A., Scott, John P., Seaborg, Kristin A., Seed, Patrick C., Serwint, Janet R., Shah, Dheeraj, Shah, Samir S., Shah, Shivang S., Shamir, Raanan, Shanti, Christina M., Shapiro, Bruce K., Shaywitz, Bennett A., Shaywitz, Sally E., Shchelochkov, Oleg A., Shulman, Stanford T., Sicherer, Scott H., Simmons, Jeffrey M., Simões, Eric A.F., Simonsen, Kari A., Simpson, Tess S., Sinclair-McBride, Keneisha R., Singh, Arunjot, Sink, Jacquelyn R., Sisk, Bryan A., Sivaraman, Vidya, Slattery, Susan M., Slavotinek, Anne M., Smith, Jessica R., Smith-Whitley, Kim, Solensky, Roland, Son, Mary Beth F., Soranno, Danielle E., Sosa, Tina K., Soto-Rivera, Carmen L., Sosinsky, Laura Stout, Souder, Emily E., Souverbielle, Cristina Tomatis, Spearman, Paul, Spiegel, David A., Spinks-Franklin, Adiaha I.A., Sprecher, Alicia J., Squires, James E., Srivastava, Siddharth, St. Geme, Joseph W., III, St. John, Rachel D., Stambough, Kathryn C., Stanberry, Lawrence R., Starke, Jeffrey R., Starr, Taylor B., Steenhoff, Andrew P., Stein, Ronen E., Steinbach, William J., Stillwell, Terri L., Stone, Deborah L., Su, Stefani, Sucato, Gina S., Suchy, Frederick J., Sullivan, Kathleen E., Swami, Sanjeev K., Szafron, Vibha A., Szilagyi, Moira, Taha, Dalal, Tan, Libo, Tantisira, Kelan G., Taylor, Alex M., Tchapyjnikov, Dmitry, Tesini, Brenda L., Theobald, Jillian L., Thielen, Beth K., Thom, Christopher S., Thornburg, Courtney D., Tieder, Joel S., Tissières, Pierre, Tolentino, Victorio R., Jr., Topjian, Alexis A., Tower, Richard L., Trachtman, Rebecca, Triebwasser, Jourdan E., Trowbridge, Sara K., Truglio, Joseph M., Tubergen, David G., Turk, Margaret A., Tymon-Rosario, Joan R., Ufberg, Paul J., Ullrich, Christina, Ullrich, Nicole, Valika, Taher S., Van Hare, George F., Van Mater, Heather A., Varnell, Charles D., Jr., Vash-Margita, Alla, Vece, Timothy J., Vemana, Aarthi P., Venditti, Charles P., Vepraskas, Sarah, Verbsky, James W., Vermilion, Jennifer A., Vickery, Brian P., Vockley, Jerry, Voynow, Judith A., Walch, Abby, Waldrop, Stephanie W., Walker, David M., Walkovich, Kelly J., Walter, Heather J., Wambach, Jennifer A., Wamithi, Susan, Wang, Julie, Wang, Marie E., Wangler, Michael F., Ware, Stephanie M., Washam, Matthew C., Wasserman, Jonathan D., Wassner, Ari J., Watson, Andrew M., Wattier, Rachel L., Weber, David R., Webster, Jennifer, Weese-Mayer, Debra E., Weinberg, Jason B., Weinman, Jason P., Weisman, Steven J., Weiss, Anna K., Weiss, Scott L., Weiss, Pamela F., Weitzman, Carol C., Wells, Lawrence, Wen, Jessica W., Wendel, Danielle R., Werlin, Steven L., Wexler, Isaiah D., Whitaker, Alexander S., White, A. Clinton, Jr., White, Perrin C., Willoughby, Rodney E., Jr., Wilschanski, Michael, Wiley, Susan E., Williams, Brendan A., Wilson, Karen M., Wilson, Pamela E., Winell, Jennifer J., Witters, Peter, Wolf, Joshua, Wolfe, Joanne, Wolfgram, Peter M., Woods, Brandon T., Wright, Benjamin L., Wright, Terry W., Wu, Eveline Y., Yagupsky, Pablo, Yang, Edward, Yang, Kesi C., Yang, Ming, Yaron, Michael, Younger, Sarah B., Yuskaitis, Christopher J., Zachariah, Philip, Zafar, Muhammad S., Zahler, Stacey G., Zajac, Lauren M., Zaky, Wafik, Zaspel, Jennifer A., Zerra, Patricia E., Zhou, Amy, Zuckerman, Barry S., and Zur, Karen B.

Published
2025
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32. An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy.

Author

Kwon, Jennifer M., Arya, Kapil, Kuntz, Nancy, Phan, Han C., Sieburg, Cory, Swoboda, Kathryn J., Veerapandiyan, Aravindhan, Assman, Beverly, Bader‐Weder, Silvia, Dickendesher, Travis L., Hansen, Jennifer, Lin, Helen, Yan, Ying, and Rao, Vamshi K.

Subjects
*SPINAL muscular atrophy, *RESPIRATORY infections, *CHILD patients
Abstract

Objective: The US risdiplam expanded access program (EAP; NCT04256265) was opened to provide individuals with Type 1 or 2 spinal muscular atrophy (SMA) who had no satisfactory treatment options access to risdiplam prior to commercial availability. The program was designed to collect safety data during risdiplam treatment. Methods: Patients were enrolled from 23 non‐preselected sites across 17 states and treated with risdiplam orally once daily. Eligible patients had a 5q autosomal recessive Type 1 or 2 SMA diagnosis, were aged ≥2 months at enrollment, and were ineligible for available and approved SMA treatments or could not continue treatment due to a medical condition, lack/loss of efficacy, or the COVID‐19 pandemic. Results: Overall, 155 patients with Type 1 (n = 73; 47.1%) or 2 SMA (n = 82; 52.9%) were enrolled and 149 patients (96.1%) completed the EAP (defined as obtaining access to commercial risdiplam, if desired). The median treatment duration was 4.8 months (range, 0.3–9.2 months). The median patient age was 11 years (range, 0–50 years), and most patients (n = 121; 78%) were previously treated with a disease‐modifying therapy. The most frequently reported adverse events were diarrhea (n = 10; 6.5%), pyrexia (n = 7; 4.5%), and upper respiratory tract infection (n = 5; 3.2%). The most frequently reported serious adverse event was pneumonia (n = 3; 1.9%). No deaths were reported. Interpretation: In the EAP, the safety profile of risdiplam was similar to what was reported in pivotal risdiplam clinical trials. These safety data provide further support for the use of risdiplam in the treatment of adult and pediatric patients with SMA. [ABSTRACT FROM AUTHOR]

Published
2022
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34. Fixed Ratio

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

Published
2013
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35. Food Intolerance

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

Published
2013
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36. Functional Connectivity

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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37. Functional Analysis

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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38. Functional Goals

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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39. Faciogenital Dysplasia

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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40. Fluoxetine

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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41. Full Physical Prompt

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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42. Facial Inversion Effect

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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43. Frontal Lobe Findings in Autism

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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44. Folk Psychology

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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45. Fragile X Syndrome

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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46. Fetal Alcohol Syndrome

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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47. Fact Memory

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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48. Fragile X Syndrome (FXS)

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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49. Functional Behavior Assessment

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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50. Face Validity

Author

Ward, Tracey, primary, Bernier, Raphael, additional, Mukerji, Cora, additional, Perszyk, Danielle, additional, McPartland, James C., additional, Johnson, Ellen, additional, Faja, Susan, additional, Nevers, Maureen, additional, Frazier, Thomas, additional, Howlin, Patricia, additional, Savage, Sarah, additional, Zane, Thomas, additional, Lanner, Traci, additional, Myers, Michelle, additional, VanBergeijk, Ernst, additional, Huestis, Samantha, additional, Bauminger-Zviely, Nirit, additional, Doehring, Peter, additional, Voorst, Guus, additional, Macy, Kelly, additional, Kwon, Jennifer M., additional, McNulty, Edward, additional, Chapman, S. Michael, additional, Crowley, Michael J., additional, Bean, Allison, additional, Hyman, Susan, additional, Scahill, Lawrence David, additional, Wing, Lorna, additional, Catania, A. Charles, additional, Thorne, John, additional, Kini, Usha, additional, Moyle, Maura, additional, Plowgian, Claire, additional, Happé, Francesca, additional, Case-Smith, Jane, additional, Schmitt, Lauren, additional, Lewis, Moira, additional, Weiss, Mary Jane, additional, Gaag, Jan Van der Rutger, additional, Hurst, Hillary, additional, Bonazinga, Laura, additional, Paul, Diane R., additional, Doyle, Carolyn A., additional, McDougle, Christopher J., additional, Perri, Kate S., additional, Moran, Marcel, additional, Stigler, Kimberly, additional, St. John, Margaret, additional, Hessl, David, additional, Schneider, Andrea, additional, Katon, Juli, additional, Hadjikhani, Nouchine, additional, Rinehart, Nicole, additional, Enticott, Peter, additional, Bradshaw, John, additional, Palmieri, Mark, additional, LaRue, Robert, additional, Molteni, John, additional, Prelock, Patricia, additional, Müller, Ralph-Axel, additional, Egan, Shaunessy, additional, Hendricks, Dawn, additional, Holman, Katherin C., additional, and D’Eramo, Kristen, additional

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2013
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