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1. Nusinersen versus sham control in later-onset spinal muscular atrophy

Author

Vogt, Sibylle, Krueger, Marcus, Pechmann, Astrid, Rippberger, Bianca, Eckenweiler, Matthias, Schara, Ulrike, Koelbel, Heike, Andres, Barbara, Rupprich, Katrin, Gangfuss, Andrea, Jachertz, Philipp, Della Marina, Adela, Sponemann, Nina, Pane, Markia, Palermo, Concetta, Piastra, Marco, Fanelli, Lavinia, de Sanctis, Roberto, Genovese, Orazio, Antonaci, Laura, Pera, Maria Carmela, Lamendola, Priscilla, Messina, Sonia, Vita, Gianluca, Di Bella, Vincenzo, Sframeli, Maria, Rosa, Matteo, Barcellona, Costanza, Distefano, Maria Grazia, Cavallaro, Filippo, Versaci, Antonio, de Luca, Francesco, Vita, Giuseppe, Nacimento Osorio, Andres, Tizzano, Eduardo, Ortez Gonzalez, Carlos Ignacio, Ortigoza Escobar, Juan Dario, Colomer Oferil, Juame, Medina Cantillo, Julita, Febrer Rotger, Anna, Vigo Morancho, Meritxell, Eldblom, Johanneh, Darin, Niklas, Kroksmark, Anna Karin, Lindstedt, Asa, Michael, Eva, Kimber, Eva, Wahlgren, Lisa, Chan, Sophelia Hoi-Shan, Chim, Stella, Chiu, Joseph, Ho, Alvin Chi Chung, Ip, Jing Kun Janice, Lam, Wendy Wai Man, Ng, Maggie Chui-San, Wan, Connie, Wong, Virginia Chun Nei, Yue, Yvonne, Arakawa, Reiko, Yamauchi, Akemi, Nagata, Satoru, Ito, Yasushi, Nakatsukasa, Hidetsugu, Takeshita, Akiko, Hirasawa, Kyoko, Ikai, Tetsuo, Eto, Kaoru, Otamni, Yui, Takeshima, Yasuhiro, Fukuda, Noroki, Tanaka, Yasuhiro, Shimomura, Hideki, Lee, Tomoko, Shibano, Takayuki, Mercuri, E., Tachikawa, Tomohiro, Darras, B. T., Chae, Jong-Hee, Chiriboga, C. A., Lim, Byung Chan, Day, J. W., Shin, Hyung-Ik, Campbell, C., Kim, Soo Yeon, Connolly, A. M., Choi, Sun Ah, Iannaccone, S. T., Son, Woo Sung, Kirschner, J., Jo, Hyemi, Kuntz, N. L., Chun, Seong Min, Saito, K., Kim, Hyuna, Shieh, P. B., Tulinius, M., Mazzone, E. S., Montes, J., Bishop, K. M., Yang, Q., Foster, R., Gheuens, S., Bennett, C. F., Farwell, W., Schneider, E., de Vivo, D. C., Finkel, R. S., Bradley, Walter G., Kaufmann, Petra, Dickson, Patricia I., Reingold, Stephen C., Davis, Charles S., Arredondo, Kristen, Castro, Diana, Cowie, Margaret, Farrow-Gillespie, Alan, Hebert, Andrew, Kauk, Melissa, Miller, Nancy, Nelson, Leslie, Spain, Thomas, Cappell, Joshua, Constantinescu, Andrei, Cruz, Rosangel, Dastgir, Jahannaz, de Vivo, Darryl, Dunaway, Sally, Engelstad, Kristin, Khandji, Alexander G., Kramer, Samantha, Marra, Jonathan, Popolizio, Molly, Salazar, Rachel, Weimer, Louis H., Aziz-Zaman, Sonya, Lamarca, Nicole, Ghosh, Partha, Al-Ghamdi, Fouad, Liew, Wendy, Graham, Robert, Berde, Charles, Sethna, Navil, Koka, Anjali, Wang, Luke, Laine, Regina, Souris, Michelle, Ordonez, Grace, Harrington, Timothy, Szelag, Heather, Pasternak, Amy, Mirek, Elizabeth, Quigley, Janet, Finkel, Richard, Berry, Debbie, Civitello, Matthew, Endsley, Julie Duke, Eden, Candace, Leon, Wendy, O'Reardon, Kathleen, Sigurdardottir, Laufey, Johnson, Craig, Turner, Jenna, Vega, Melisa, Weber-Guzman, Fabiola, Zinn, Matthias, Rocha, Ana Carolina Tesi, Watson, Karolina, d'Souza, Genevieve, Ramamurthi, R. J., Gee, Richard, Kitsuwa-Lowe, Janis, Hagerman, Katharine, Crasta, Sheela, Welsh, Lesly, Paulose, Shirley, Mcfall, Danielle, Perez, Jennifer, Patnaik, Swetapadma, Sanjanwala, Bharati, Sakamuri, Sarada, Proud, Crystal, Purse, Bona Park, Duong, Trinh Tina, Sampson, Jacinda, Tennekoon, Gihan, Brandsema, John, Glanzman, Allan, Flickinger, Jean, Toms, Michele, Adang, Laura, Stanford, Delores, Mayer, Oscar, Zigmont, Joshua, Chadehumbe, Madeline, Kichula, Elizabeth, Finanger, Erika, Russman, Barry, Roberts, Colin, Frank, Andrea, Benjamin, Danielle, Zilke, Kirsten, Golumbek, Paul T., Zaidman, Craig M., Anand, Pallavi, Gadeken, Rebecca, Siener, Catherine, Kuntz, Nancy, Epstein, Leon, Krueger, Jena, Goldman, Stewart, Krosschell, Kristin, Blomgren, Colleen, Choi, Hyoung Won, Kurz, Jonathan, Parsons, Julie, Janas, Joanne, Yang, Michele, Ballard, Alison, Carry, Terri, Shea, Stephanie, Bielsky, Alan, Booker, Kaylee, Camuto, Alicia, Lord-Halvorson, Sierra, Gibbons, Melissa, Zimmerman, Carl, Allen, Victoria, Fuhr, Peter, Johnson, Hannah, Tran, Vi, Vanderveen, Gina, Shieh, Perry, Fowler, Eileen, Parziale, Nicholas, Rao, Lekha, Skura, Christy, Kelley, Carolyn, Shu, Francy, Oskoui, Maryam, Zielinski, David, Poulin, Chantal, Ingelmo, Pablo Mauricio, Desilets, Sarah Turgeon, Dinunzio, Pamela, Rivera, Gonzalo, Srour, Myriam, Arpin, Stephanie, Goobie, Sharan, Gibson, Paul, Scholtes, Cheryl, Mcdonald, Wendy, Zapata, Eugenio, Nguyen, Cam-Tu Emilie, Servais, Laurent, Gargaun, Elena, Le Moing, Anne-Gaelle, Gidaro, Teresa, Vialle, Raphael, Guye, Marie-Laurence, Lilien, Charlotte, Olliver, Gwenn, Gilabert, Stephanie, Borell, Sabine, Wider, Sabine, Stein, Sabine, Universität Duisburg-Essen = University of Duisburg-Essen [Essen], Department of Paediatrics, Università cattolica del Sacro Cuore = Catholic University of the Sacred Heart [Roma] (Unicatt), Vall d'Hebron University Hospital [Barcelona], CIBER de Enfermedades Raras (CIBERER), Handicaps génétiques de l'enfant (Inserm U393), Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), Seoul National University Hospital, Max-Planck-Institut für Mikrostrukturphysik (MPI-HALLE), Max-Planck-Gesellschaft, The University of Tokyo (UTokyo), Institut de Chimie Organique et Analytique (ICOA), Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Université d'Orléans (UO)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut de Chimie du CNRS (INC)-Centre National de la Recherche Scientifique (CNRS), Institute of Plasma Physics, Chinese Academy of Sciences (ASIPP), Institut Necker Enfants-Malades (INEM - UM 111 (UMR 8253 / U1151)), Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université Paris Cité (UPCité), Laboratoire de mécanique des solides (LMS), École polytechnique (X)-Mines Paris - PSL (École nationale supérieure des mines de Paris), Université Paris sciences et lettres (PSL)-Université Paris sciences et lettres (PSL)-Centre National de la Recherche Scientifique (CNRS), CureSMA [Elk Grove Village, IL, USA], Harvard Medical School [Boston] (HMS), Institute for Marine and Antarctic Studies [Hobart] (IMAS), University of Tasmania [Hobart, Australia] (UTAS), The Hospital for sick children [Toronto] (SickKids), University of Toronto, The Open University [Milton Keynes] (OU), Department of Pediatrics, Feinberg School of Medicine, Northwestern University [Evanston]-Northwestern University [Evanston]-Northwestern University, University Hospital Basel [Basel], McGill University Health Center [Montreal] (MUHC), Service de génétique [Tours], Centre Hospitalier Régional Universitaire de Tours (CHRU Tours)-Hôpital Bretonneau, Service of Clinical Trials and Databases, Institut de Myologie, Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Association française contre les myopathies (AFM-Téléthon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS)-Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Association française contre les myopathies (AFM-Téléthon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Association française contre les myopathies (AFM-Téléthon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), CHU Amiens-Picardie, Groupe de Recherche sur l'Analyse Multimodale de la Fonction Cérébrale - UMR INSERM_S 1105 (GRAMFC), Université de Picardie Jules Verne (UPJV)-CHU Amiens-Picardie-Institut National de la Santé et de la Recherche Médicale (INSERM), CHU Trousseau [APHP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), University of Oxford, Schara, Ulrike (Beitragende*r), Koelbel, Heike (Beitragende*r), Rupprich, Katrin (Beitragende*r), Gangfuss, Andrea (Beitragende*r), Della Marina, Adela (Beitragende*r), and Sponemann, Nina (Beitragende*r)

Subjects
0301 basic medicine, Male, [SDV]Life Sciences [q-bio], spinal, Medizin, Oligonucleotides, Spinal Muscular Atrophies of Childhood, Pediatrics, law.invention, Age of Onset, Child, Child, Preschool, Double-Blind Method, Female, Humans, Infant, Injections, Spinal, Least-Squares Analysis, Motor Skills, Oligonucleotides, Antisense, Medicine (all), 0302 clinical medicine, age of onset, Randomized controlled trial, law, Clinical endpoint, inglese, injections, Motor skill, motor skills, General Medicine, SMA, Settore MED/26 - NEUROLOGIA, medicine.anatomical_structure, female, Anesthesia, Nusinersen, Spinal, antisense, preschool, Injections, 03 medical and health sciences, least-squares analysis, Settore MED/39 - NEUROPSICHIATRIA INFANTILE, Settore MED/41 - ANESTESIOLOGIA, medicine, Antisense, Preschool, business.industry, Spinal muscular atrophy, Motor neuron, medicine.disease, 030104 developmental biology, Age of onset, business, 030217 neurology & neurosurgery
Abstract

Background: Nusinersen is an antisense oligonucleotide drug that modulates pre–messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA). Methods: We conducted a multicenter, double-blind, sham-controlled, phase 3 trial of nusinersen in 126 children with SMA who had symptom onset after 6 months of age. The children were randomly assigned, in a 2:1 ratio, to undergo intrathecal administration of nusinersen at a dose of 12 mg (nusinersen group) or a sham procedure (control group) on days 1, 29, 85, and 274. The primary end point was the least-squares mean change from baseline in the Hammersmith Functional Motor Scale–Expanded (HFMSE) score at 15 months of treatment; HFMSE scores range from 0 to 66, with higher scores indicating better motor function. Secondary end points included the percentage of children with a clinically meaningful increase from baseline in the HFMSE score (≥3 points), an outcome that indicates improvement in at least two motor skills. Results: In the prespecified interim analysis, there was a least-squares mean increase from baseline to month 15 in the HFMSE score in the nusinersen group (by 4.0 points) and a least-squares mean decrease in the control group (by –1.9 points), with a significant between-group difference favoring nusinersen (least-squares mean difference in change, 5.9 points; 95% confidence interval, 3.7 to 8.1; P Conclusions: Among children with later-onset SMA, those who received nusinersen had significant and clinically meaningful improvement in motor function as compared with those in the control group. (Funded by Biogen and Ionis Pharmaceuticals; CHERISH ClinicalTrials.gov number, NCT02292537. opens in new tab.)

Published
2018

2. VP.56a Real-world assessment of onasemnogene abeparvovec treatment in patients with spinal muscular atrophy: RESTORE/post-marketing surveillance in Japan.

Author

Saito, K., Nagao, R., Tsuchida, K., Teshima, R., and Kawase, K.

Subjects
*SPINAL muscular atrophy, *ACUTE kidney failure, *JAPANESE people
Abstract

Availability of real-world data on spinal muscular atrophy (SMA) has grown, but region-specific data evaluating specific treatments and outcomes within discrete national health systems are limited. We assessed treatment patterns and long-term, real-world outcomes for patients with SMA treated with onasemnogene abeparvovec (OA) in Japan. In line with Japanese regulatory requirements, post-marketing surveillance (PMS) was conducted for patients treated with OA as part of RESTORE, a real-world, prospective, multicenter, multinational, observational registry of patients with SMA. We collected and evaluated data on patients enrolled in RESTORE/PMS who received OA in Japan. This report provides interim results from May 18, 2021 (PMS start) to May 23, 2021 (data cutoff) for 19 patients that had been treated with OA (age range at infusion, 0.0, 24.0 months; weight range at infusion, 2.7, 10.1 kg). Seven patients (36.8%) received OA monotherapy, and 12 (63.2%) received OA after initial nusinersen. Patients had two (n=11/19, 57.9%) or three (n=8/19, 42.1%) copies of the SMN2 gene. Of 5 patients with ≥2 CHOP INTEND assessments, 4 improved (3 achieved a ≥4-point increase), and 1 had a 1-point decrease (change per month range, –0.14, +3.42 points). Of 4 patients with ≥2 motor milestone assessments, 2 achieved new milestones, 1 maintained milestones, and 1 recorded no milestone achievements. No loss of achieved milestones was reported after OA. All patients reported at least 1 treatment-emergent adverse event (AE). Three patients (15.8%) experienced at least 1 serious AE related to OA. Thrombotic microangiopathy and associated events (hemolysis and acute kidney injury) were reported for 1 patient. No deaths were reported. To date, patients in Japan enrolled in RESTORE/PMS achieved or maintained motor milestones after OA. The AE experience of OA for Japanese patients is consistent with the overall safety profile of OA, with no new safety concerns identified. [ABSTRACT FROM AUTHOR]

Published
2022
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3. P.114 SUNFISH parts 1 and 2: 3-year efficacy and safety of risdiplam in types 2 and 3 spinal muscular atrophy (SMA).

Author

Day, J., Deconinck, N., Mazzone, E., Nascimento, A., Oskoui, M., Saito, K., Vuillerot, C., Baranello, G., Boespflug-Tanguy, O., Goemans, N., Kirschner, J., Kostera-Pruszczyk, A., Servais, L., Braid, J., Gerber, M., Gorni, K., Martin, C., Scalco, R., Yeung, W., and Mercuri, E.

Subjects
*SPINAL muscular atrophy, *MOTOR neurons
Abstract

Spinal muscular atrophy (SMA) affects individuals with a broad age range and spectrum of disease severity. Risdiplam (EVRYSDI®) is a centrally and peripherally distributed, oral survival of motor neuron 2 (SMN2) pre-mRNA splicing modifier that has been approved in more than 70 countries worldwide. SUNFISH (NCT02908685) is a multicenter, two-part, randomized, placebo-controlled, double-blind study in patients with Types 2 and 3 SMA (inclusion criteria: 2–25 years at enrollment). Part 1 (N=51) assessed the safety, tolerability and pharmacokinetics/pharmacodynamics of different risdiplam dose levels in patients with Types 2 and 3 SMA (ambulant and non-ambulant). Part 2 (N=180) assessed the efficacy and safety of the Part 1-selected dose of risdiplam versus placebo in Type 2 and non-ambulant type 3 SMA. In part 2, participants were treated with risdiplam or placebo for 12 months; all participants then received risdiplam until month 24. At month 24, patients were offered the opportunity to enter the open-label extension phase. The primary outcome of Part 2 was met, showing a statistically significant difference in the change from baseline in 32-item motor function measure total score at month 12 in patients treated with risdiplam (n=120) versus placebo (n=60). The gains observed with risdiplam treatment at month 12 were maintained between months 12 and 36. At month 36, there were no treatment-related safety findings leading to withdrawal. Here we present 36-month efficacy and safety data from SUNFISH. SUNFISH is ongoing and will provide further long-term efficacy and safety data of risdiplam in a broad population of children, teenagers and adults with SMA. [ABSTRACT FROM AUTHOR]

Published
2022
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4. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.

Author

Finkel, R. S., Tizzano, E., Topaloglu, H., Tulinius, M., Glanzman, A. M., Bishop, K., Bennett, C. F., Schneider, E., Zhong, Z. J., Gheuens, S., Farwell, W., De Vivo, D. C., Mercuri, E., Darras, B. T., Connolly, A. M., Kuntz, N. L., Kirschner, J., Chiriboga, C. A., Montes, J., and Saito, K.

Subjects
*SPINAL muscular atrophy, *MOTOR neurons, *RNA splicing, *NEUROMUSCULAR diseases, *HUMAN deletion mutation, *RNA metabolism, *AGE factors in disease, *ARTIFICIAL respiration, *CARRIER proteins, *COMPARATIVE studies, *SPINAL injections, *RESEARCH methodology, *MEDICAL cooperation, *MOTOR ability, *MUSCULAR atrophy, *NUCLEOTIDES, *PROGNOSIS, *RESEARCH, *RNA, *STATISTICAL sampling, *SURVIVAL analysis (Biometry), *EVALUATION research, *RANDOMIZED controlled trials, *BLIND experiment, *THERAPEUTICS
Abstract

Background: Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an antisense oligonucleotide drug that modifies pre-messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein.Methods: We conducted a randomized, double-blind, sham-controlled, phase 3 efficacy and safety trial of nusinersen in infants with spinal muscular atrophy. The primary end points were a motor-milestone response (defined according to results on the Hammersmith Infant Neurological Examination) and event-free survival (time to death or the use of permanent assisted ventilation). Secondary end points included overall survival and subgroup analyses of event-free survival according to disease duration at screening. Only the first primary end point was tested in a prespecified interim analysis. To control the overall type I error rate at 0.05, a hierarchical testing strategy was used for the second primary end point and the secondary end points in the final analysis.Results: In the interim analysis, a significantly higher percentage of infants in the nusinersen group than in the control group had a motor-milestone response (21 of 51 infants [41%] vs. 0 of 27 [0%], P<0.001), and this result prompted early termination of the trial. In the final analysis, a significantly higher percentage of infants in the nusinersen group than in the control group had a motor-milestone response (37 of 73 infants [51%] vs. 0 of 37 [0%]), and the likelihood of event-free survival was higher in the nusinersen group than in the control group (hazard ratio for death or the use of permanent assisted ventilation, 0.53; P=0.005). The likelihood of overall survival was higher in the nusinersen group than in the control group (hazard ratio for death, 0.37; P=0.004), and infants with a shorter disease duration at screening were more likely than those with a longer disease duration to benefit from nusinersen. The incidence and severity of adverse events were similar in the two groups.Conclusions: Among infants with spinal muscular atrophy, those who received nusinersen were more likely to be alive and have improvements in motor function than those in the control group. Early treatment may be necessary to maximize the benefit of the drug. (Funded by Biogen and Ionis Pharmaceuticals; ENDEAR ClinicalTrials.gov number, NCT02193074 .). [ABSTRACT FROM AUTHOR]

Published
2017
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6. P.104 Treatments and outcomes for patients with spinal muscular atrophy (SMA) type 2: Findings from RESTORE registry.

Author

Servais, L., Benguerba, K., De Vivo, D., Kirschner, J., Muntoni, F., Proud, C., Tizzano, E., Quijano-Roy, S., Desguerre, I., Saito, K., Raju, D., LaMarca, N., Sun, R., Anderson, F., Faulkner, E., and Finkel, R.

Subjects
*SPINAL muscular atrophy, *TREATMENT effectiveness, *MEDICAL registries, *GENE therapy
Abstract

We aimed to describe real-world treatment patterns/outcomes for patients with SMA type 2 (SMA2). RESTORE is an ongoing SMA patient registry. We analyzed changes in motor milestones and disability scores and assessed treatment-emergent adverse events (TEAEs) for patients who received ≥1 disease-modifying therapy (DMT; gene therapy [onasemnogene abeparvovec (OA)] or SMN2 modulator [nusinersen/risdiplam]). As of Nov. 23, 2021, RESTORE included 48 patients with SMA2, and 29 (60.4%) were female. Eight (16.7%), 37 (77.1%), 2 (4.2%) and 1 (2.1%) had 2, 3, 4 and ≥4 SMN2 gene copies, respectively. Median age at SMA diagnosis was 14.0 months and at first DMT administration, 16.0 months. Median interval between diagnosis and first DMT was 1 month. Median age at first DMT differed between groups (p=0.0033): OA monotherapy, 16.5 months (n=24); nusinersen monotherapy, 25 months (n=10); risdiplam monotherapy, 8 months (n=1); switching from other DMT to OA, 12 months (n=11); and receiving add-on therapy to OA, 15.5 months (n=2). Of 26 patients with ≥2 motor milestone assessments (≥1 post-DMT administration), all but 4 (OA only [n=3], nusinersen only [n=1]) maintained or achieved new milestones: 13 (59.1%) received OA monotherapy; 6 (27.3%) switched from nusinersen to OA; 1 (4.5%) received add-on to OA; and 2 (9.1%) received nusinersen only. Median HFMSE score change was 5.5 (n=10), with median monthly change of 0.6 (n=10) for all patients, and 11.0 (n=5), with median monthly change of 0.7 (n=6) for patients receiving OA monotherapy. Any-grade TEAEs were recorded for 12/24 (50%) patients who received OA monotherapy, 7/11 (63.6%) who switched from nusinersen to OA, and 1/2 (50%) who received add-on treatment to OA. Of patients who received OA, 3 required non-oral feeding support and 2 required BiPAP ventilation. AEs in RESTORE are consistent with OA experience. No new safety signals were identified. OA is effective and has an acceptable safety profile for patients with SMA2. [ABSTRACT FROM AUTHOR]

Published
2022
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9. SMA - TREATMENT: EP.284 SUNFISH Part 2: 24-month efficacy and safety of risdiplam in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA).

Author

Nascimento, A., Day, J., Deconinck, N., Mazzone, E., Oskoui, M., Saito, K., Vuillerot, C., Baranello, G., Boespflug-Tanguy, O., Goemans, N., Kirschner, J., Kostera-Pruszczyk, A., Servais, L., Gerber, M., Gorni, K., Martin, C., Scalco, R., Staunton, H., Yeung, W., and Mercuri, E.

Subjects
*SPINAL muscular atrophy, *PATIENT safety
Published
2021
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10. SMA - TREATMENT: EP.274 Onasemnogene Abeparvovec Gene Therapy in Presymptomatic spinal muscular atrophy (SMA): SPR1NT study update in children with 3 Copies of SMN2.

Author

Strauss, K., Muntoni, F., Farrar, M., Saito, K., Mendell, J., Servais, L., McMillan, H., Swoboda, K., Kwon, J., Zaidman, C., Chiriboga, C., Iannaccone, S., Krueger, J., Parsons, J., Shieh, P., Kavanagh, S., Chand, D., Tauscher-Wisniewski, S., and Macek, T.

Subjects
*SPINAL muscular atrophy, *GENE therapy
Published
2021
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11. SMA CLINICAL DATA: EP.249 ANCHOVY: A retrospective cohort study of the natural history of type 1 spinal muscular atrophy (SMA) using medical record data.

Author

Zanoteli, E., Cances, C., Vlodavets, D., Comi, G., Masson, R., Mazurkiewicz-Bełdzińska, M., Saito, K., Dodman, A., El-Khairi, M., Gorni, K., Gravestock, I., Hoffart, J., Scalco, R., and Darras, B.

Subjects
*SPINAL muscular atrophy, *DATA recorders & recording, *COHORT analysis, *ANCHOVIES, *RETROSPECTIVE studies
Published
2021
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