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146 results on '"Kaye, Edward M."'

1. List of Contributors

Author

Aaen, Gregory, primary, Abroms, Israel F., additional, Ådén, Ulrika, additional, Ahlsten, Gunnar, additional, Aird, Robert B., additional, Al-Zaidy, Samiah A., additional, Andermann, Fred, additional, Anlar, Banu, additional, Arzimanoglou, Alexis, additional, Ashwal, Stephen, additional, Augustine, Erika, additional, Ballaban-Gil, Karen, additional, Bamford, Nigel S., additional, Barlow, Charles F., additional, Bast, Thomas, additional, Bates, David, additional, Baumann, Robert J., additional, Bertini, Enrico, additional, Beya, Alidor, additional, Blaw, Michael, additional, Bodensteiner, John, additional, Bonthius, Daniel J., additional, Brin, Amy E., additional, Brockmann, Knut, additional, Brown, John Keith, additional, Brown, Stuart B., additional, Brumback, Audrey Christine, additional, Bureau, Michelle, additional, Burke, James R., additional, Bye, Annie, additional, Camfield, Carol, additional, Camfield, Peter, additional, Campistol Plana, Jaume, additional, Canale, Dee James, additional, Caneris, Onasis, additional, Caraballo, Roberto H., additional, Caviness, Alison Chantal, additional, Chao, Hsiao-Tuan, additional, Chapman, Catherine A., additional, Chaves-Carballo, Enrique, additional, Cho, Yoon-Jae, additional, Christen, Hans-Jürgen, additional, Chugani, Harry T., additional, Cioni, Giovanni, additional, Clark, David, additional, Cliff, Edward Robert Scheffer, additional, Cochran, Frederick B., additional, Cohen, Bruce H., additional, Cohen, Maynard M., additional, Collins, Kevin, additional, Covanis, Athanasios, additional, Critchley, Macdonald, additional, Cross, J. Helen, additional, Crumrine, Patricia K., additional, Curatolo, Paolo, additional, Davies, Pamela A., additional, deVeber, Gabrielle, additional, De Vivo, Darryl C., additional, de Vries, Linda S., additional, De Waele, Liesbeth, additional, DeMyer, William, additional, Devlin, Anita, additional, Dobyns, William B., additional, Dodson, W. Edwin, additional, Donald, Kirsty, additional, Duffy, Frank H., additional, Dunn, David W., additional, Dunn, Henry G., additional, Dure, Leon S., additional, Dyken, Paul Richard, additional, Encha-Razavi, Férechté, additional, Erenberg, Gerald, additional, Estes, Melinda L., additional, Evrard, Philippe, additional, Ferriero, Donna, additional, Ferry, Peggy, additional, Fine, Archie, additional, Fine, Edward J., additional, Fine, John S., additional, Finkel, Richard S., additional, Fischer, Alain, additional, Fischer, Christine, additional, Fogan, Lance, additional, Fowler, Glenn W., additional, Frank, Yitzchak, additional, Fullerton, Heather J., additional, Furukawa, Tetsuo, additional, Gabriel, Ronald S., additional, Galanopoulou, Aristea S., additional, Gardner-Medwin, David, additional, Garg, Bhuwan, additional, Genton, Pierre, additional, George, Mark S., additional, Gineste, Thierry, additional, Giza, Christopher C., additional, Goemans, Nathalie, additional, Golden, Gerald S., additional, Golden, Jeffrey Alan, additional, Goldstein, Gary W., additional, Gomez, Christopher, additional, Gomez, Manuel R., additional, Gomez, Timothy, additional, Goodkin, Howard P., additional, Gordon, Neil, additional, Gressens, Pierre, additional, Groger, Helmut, additional, Guerrini, Renzo, additional, Gurnett, Christina A., additional, Gussoni, Emanuela, additional, Haas, Richard, additional, Hagberg, Bengt, additional, Haller, Jerome S., additional, Hartman, Adam L., additional, Haruda, Fred, additional, Hirtz, Deborah, additional, Hogan, Gwendolyn R., additional, Hunt, Guy M., additional, Iannaccone, Susan T., additional, Eleanor Inder, Terrie, additional, Ionasescu, Victor, additional, Jansen, Katrien, additional, Jiang, Yuwu, additional, Kaminski, Henry J., additional, Kamoshita, Shigehiko, additional, Kang, Peter B., additional, Kaufman, David M., additional, Kaufmann, Walter E., additional, Kaye, Edward M., additional, Kellaway, Peter, additional, Kelley, Rhona S., additional, Kennedy, Charles, additional, Kim, Young-Min, additional, Kirby, Michael, additional, Kirton, Adam, additional, Kobayashi, Eliane, additional, Kossoff, Eric H., additional, Koutroumanidis, Michail, additional, Krupp, Lauren, additional, Lange, Bernadette M., additional, Lanska, Douglas J., additional, Lanska, Mary Jo, additional, Larsen, Paul D., additional, Lassoff, Samuel J., additional, Laterra, John, additional, Lemieux, Bernard, additional, Lenn, Nicholas J., additional, Logan, William J., additional, Lomax, Elizabeth, additional, Longo, Lawrence D., additional, Lorris Betz, A., additional, Manyam, Bala V., additional, Marks, Warren A., additional, Massey, E. Wayne, additional, Mate, Laszlo J., additional, McKinlay, Ian, additional, McLean, William T., additional, McLellan, Ailsa, additional, Mehler, Mark F., additional, Melchior, Johannes C., additional, Michelson, David J., additional, Miller, Steven P., additional, Miller, Suzanne L., additional, Millichap, J. Gordon, additional, Minns, Robert A., additional, Mizrahi, Eli M., additional, Moser, Ann B., additional, Moshé, Solomon L., additional, Muhle, Hiltrud, additional, Muntoni, Francesco, additional, Naidu, Sakkubai, additional, Narayanan, Vinodh, additional, Nardocci, Nardo, additional, Neil, Jeffrey J., additional, Neumeyer, Ann, additional, Noetzel, Michael J., additional, Nomura, Yoshiko, additional, Nordli, Douglas R., additional, North, Kathryn, additional, Ohtsuka, Yoko, additional, O’Callaghan, Finbar J.K., additional, Packer, Roger J., additional, Pastores, Gregory M., additional, Patterson, Marc C., additional, Pearl, Phillip L., additional, Philippart, Michel, additional, Pihko, Helena S., additional, Piller, Gordon, additional, Platz, Thomas F., additional, Poduri, Annapurna, additional, Pollack, Michael A., additional, Porter, Brenda E., additional, Provis, Michèle, additional, Rating, Dietz, additional, Reich, Harold, additional, Remler, Bernd, additional, Rho, Jong M., additional, Richards, Peter, additional, Richardson, Edward P., additional, Richardson, Sylvia O., additional, Roach, E. Steve, additional, Rose, Arthur L., additional, Rozear, Marvin P., additional, Rubinstein, Lucien J., additional, Rust, Robert S., additional, Saini, Arushi Gahlot, additional, Saint-Anne Dargassies, Suzanne, additional, Sarnat, Harvey B., additional, Sarwar, Mohammad, additional, Satran, Richard, additional, Schneider, Sanford, additional, Schrank, Waltraud, additional, Scott, Rodney C., additional, Seinfeld, Syndi, additional, Selcen, Duygu, additional, Sestan, Nenad, additional, Shapiro, Steven, additional, Sherr, Elliott H., additional, Shevell, Michael, additional, Shield, Lloyd, additional, Sidman, Richard L., additional, Silverstein, Faye S., additional, Sinnreich, Michael, additional, Snead, O. Carter, additional, Solomons, Regan, additional, Soria-Duran, Emilio, additional, Stafstrom, Carl E., additional, Steven Roach, E., additional, Stevens, Harold, additional, Strassburg, Hans Michael, additional, Stumpf, David A., additional, Sullivan, Thomas, additional, Swick, Herbert M., additional, Swisher, Charles N., additional, Takahashi, Takao, additional, Tein, Ingrid, additional, Tochen, Laura, additional, Thomas, Eva E., additional, Thompson, Alan, additional, Toor, Svinder S., additional, Tyler, H. Richard, additional, Uldall, Peter, additional, Urion, David K., additional, Valappil, Ahsan Moosa Naduvil, additional, Van Toorn, Ronald, additional, Vermilion, Jennifer, additional, Vidaver, Doris, additional, Vohr, Betty R., additional, Vollmer, Brigitte, additional, Volpe, Joseph J., additional, Waber, Deborah P., additional, Wainwright, Mark S., additional, Waites, Lucius, additional, Walsh, Christopher, additional, Weindl, Adolf, additional, Whelan, Mary Anne, additional, White, Larry E., additional, Whittemore, Vicky Holets, additional, Wilmshurst, Jo, additional, Wirrell, Elaine, additional, Wolf, Nicole I., additional, Youssef, Paul, additional, Zempel, John, additional, Zoghbi, Huda Y., additional, Zuberi, Sameer M., additional, and Zupanc, Mary, additional

Published
2021
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7. Eteplirsen for the treatment of Duchenne muscular dystrophy

Author

Mendell, Jerry R., Rodino-Klapac, Louise R., Sahenk, Zarife, Roush, Kandice, Bird, Loren, Lowes, Linda P., Alfano, Lindsay, Gomez, Ann Maria, Lewis, Sarah, Kota, Janaiah, Malik, Vinod, Shontz, Kim, Walker, Christopher M., Flanigan, Kevin M., Corridore, Marco, Kean, John R., Allen, Hugh D., Shilling, Chris, Melia, Kathleen R., Sazani, Peter, Saoud, Jay B., and Kaye, Edward M.

Published
2013
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8. Newborn screening by tandem mass spectrometry for medium-chain acyl-CoA dehydrogenase deficiency: a cost-effectiveness analysis

Author

Venditti, Laura N., Venditti, Charles P., Berry, Gerard T., Kaplan, Paige B., Kaye, Edward M., Glick, Henry, and Stanley, Charles A.

Abstract

Objective. To determine whether newborn screening by tandem mass spectrometry (MS/MS) for medium-chain acyl-CoA dehydrogenase deficiency (MCADD) is cost-effective versus not screening and to define the contributions of disease, test, and population parameters on the decision. Methods. A decision-analytic Markov model was designed to perform cost-effectiveness and cost-utility analyses measuring the discounted, incremental cost per life-year saved and per quality-adjusted life-year saved of newborn screening for MCADD compared with not screening. A hypothetical cohort of neonates made transitions among a set of health states that reflected clinical status, morbidity, and cost. Outcomes were estimated for time horizons of 20 and 70 years. Probabilities and costs were derived from a retrospective chart review of a 32-patient cohort treated over the past 30 years at the Children's Hospital of Philadelphia, clinical experience with MCADD patient management, patient-family interviews, cost surveys, state sources, and published studies. In addition to older patients who came to medical attention by symptomatic presentation, our patient group included 6 individuals whose MCADD had been diagnosed by supplemental newborn screening. Estimates of the expected net changes in costs and life expectancy for MCADD screening were used to compute the incremental cost-effectiveness ratios. Sensitivity analyses were performed on key input variables, and 95% confidence intervals (CIs) were computed through second-order Monte Carlo simulations. Results. In our base-case analysis over the first 20 years of life, the cost of newborn screening for MCADD was approximately $11 000 (2001 US dollars; 95% CI: Conclusions. Simulation modeling indicates that newborn screening for MCADD reduces morbidity and mortality at an incremental cost below the range for accepted health care interventions. At the 70-year horizon, the model predicts that almost all of the additional costs of screening would be offset by avoided sequelae. Pediatrics 2003;112:1005-1015; cost-utility, cost-effectiveness, newborn screening, inborn errors of metabolism, MCADD., ABBREVIATIONS. IEM, inborn error of metabolism; MS/MS, tandem mass spectrometry; MCADD, medium-chain acyl-CoA dehydrogenase deficiency; PKU, phenylketonuria; QALY, quality-adjusted life-year; USD, US dollars; CI, confidence interval. Inborn errors of metabolism [...]

Published
2003

13. A controlled trial of diazepam administered during febrile illnesses to prevent recurrence of febrile seizures

Author

Rosman, N. Paul, Colton, Theodore, Labazzo, Jan, Gilbert, Paula L., Gardella, Nancy B., Kaye, Edward M., Van Bennekom, Carla, and Winter, Michael R.

Subjects
Diazepam -- Health aspects, Convulsions in children -- Prevention, Febrile convulsions -- Prevention
Abstract

Diazepam may be a safe and effective therapy for preventing febrile seizures in children. Febrile seizures can cause brain injuries. Children between the ages of six months and five years were randomly assigned to receive either diazepam or a placebo at the onset of a fever. Diazepam treatment was assigned to 202 children and the placebo was assigned to 204. Parents were instructed to give the drug at the first sign of fever and to continue treatment until the child had been without a fever for 24 hours. Seven children in the diazepam group had seizures, compared with 29 children in the placebo group. This is an 82% reduction in the risk of seizures in the children taking diazepam. Most side effects from the drug were mild and some lessened when the dose was reduced.

Published
1993

19. Longitudinal effect of eteplirsen vs. historical control on ambulation in DMD

Author

Mendell, Jerry R, Goemans, Nathalie, Lowes, Linda P, Alfano, Lindsay N, Berry, Katherine, Shao, James, Kaye, Edward M, Mercuri, Eugenio, Pane, M, Mazzone, E, Messina, S, Vita, Gl, D'Amico, A, Berardinelli, A, Torrente, Y, Magri, F, Comi, Gp, Baranello, G, Mongini, Tiziana Enrica, Pini, A, Battini, R, Pegoraro, E, Bruno, C, Politano, L, Previtali, S, Hamid, Ha, Byrne, Bj, Connolly, Am, Dracker, Ra, Frank, Lm, Heydemann, Pt, O'Brien, Kc, Sparks, Se, Specht, La, Rodino Klapac, L, Sahenk, Z, Al Zaidy, S, Cripe, Lh, and Lewis, S.

Subjects
2'O-methyl-ribonucleoside phosphorothioate, 6MWT, Duchenne muscular dystrophy (DMD), eteplirsen, phosphorodiamidate morpholino oligomer (PMO), exon skipping, historical control
Published
2015

20. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

Author

Mendell, Jerry R, Goemans, Nathalie, Lowes, Linda P, Alfano, Lindsay N, Berry, Katherine, Shao, Jame, Kaye, Edward M, Mercuri, Eugenio Maria, Mercuri, Eugenio Maria (ORCID:0000-0002-9851-5365), Mendell, Jerry R, Goemans, Nathalie, Lowes, Linda P, Alfano, Lindsay N, Berry, Katherine, Shao, Jame, Kaye, Edward M, Mercuri, Eugenio Maria, and Mercuri, Eugenio Maria (ORCID:0000-0002-9851-5365)

Abstract

To continue evaluation of the long-term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer designed to skip DMD exon 51 in patients with Duchenne muscular dystrophy (DMD). Three-year progression of eteplirsen-treated patients was compared to matched historical controls (HC). METHODS: Ambulatory DMD patients who were ≥7 years old and amenable to exon 51 skipping were randomized to eteplirsen (30/50mg/kg) or placebo for 24 weeks. Thereafter, all received eteplirsen on an open-label basis. The primary functional assessment in this study was the 6-Minute Walk Test (6MWT). Respiratory muscle function was assessed by pulmonary function testing (PFT). Longitudinal natural history data were used for comparative analysis of 6MWT performance at baseline and months 12, 24, and 36. Patients were matched to the eteplirsen group based on age, corticosteroid use, and genotype. RESULTS: At 36 months, eteplirsen-treated patients (n = 12) demonstrated a statistically significant advantage of 151m (p < 0.01) on 6MWT and experienced a lower incidence of loss of ambulation in comparison to matched HC (n = 13) amenable to exon 51 skipping. PFT results remained relatively stable in eteplirsen-treated patients. Eteplirsen was well tolerated. Analysis of HC confirmed the previously observed change in disease trajectory at age 7 years, and more severe progression was observed in patients with mutations amenable to exon skipping than in those not amenable. The subset of patients amenable to exon 51 skipping showed a more severe disease course than those amenable to any exon skipping. INTERPRETATION: Over 3 years of follow-up, eteplirsen-treated patients showed a slower rate of decline in ambulation assessed by 6MWT compared to untreated matched HC. Ann Neurol 2016;79:257-271.

Published
2016

23. Neurogenetics

Author

Kaye, Edward M.

Subjects
Neurogenetics (Book), Books -- Book reviews
Published
2000

24. Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

Author

Passini, Marco A., primary, Bu, Jie, additional, Richards, Amy M., additional, Kinnecom, Cathrine, additional, Sardi, S. Pablo, additional, Stanek, Lisa M., additional, Hua, Yimin, additional, Rigo, Frank, additional, Matson, John, additional, Hung, Gene, additional, Kaye, Edward M., additional, Shihabuddin, Lamya S., additional, Krainer, Adrian R., additional, Bennett, C. Frank, additional, and Cheng, Seng H., additional

Published
2011
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42. Childhood ataxia with diffuse central nervous system hypomyelination

Author

Schiffmann, Raphael, primary, Moller, Johanna R., additional, Trapp, Bruce D., additional, Shih, Henry H-L., additional, Farrer, Robert G., additional, Katz, David A., additional, Alger, Jeffry R., additional, Parker, Colette C., additional, Hauer, Peter E., additional, Kaneski, Christine R., additional, Heiss, John D., additional, Kaye, Edward M., additional, Quarles, Richard H., additional, Brady, Roscoe O., additional, and Barton, Norman W., additional

Published
1994
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44. Dysmyelinogenesis in animal model of GM1 gangliosidosis

Author

Kaye, Edward M., primary, Alroy, Joseph, additional, Raghavan, Srinivasa S., additional, Schwarting, Gerald A., additional, Adelman, Lester S., additional, Runge, Val, additional, Gelblum, Dafna, additional, Thalhammer, Johann G., additional, and Zuniga, Gonzalo, additional

Published
1992
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