| Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam (NCT05156320) | Scholar Rock, Inc. | 2022-04-14 |
| Newborn Screening for Spinal Muscular Atrophy (NCT05481164) | University of Oxford | 2022-03-08 |
| Procedural Sedation for Pediatric Patients With Spinal Muscular Atrophy (NCT05291962) | Ankara City Hospital Bilkent | 2022-01-01 |
| A BHI to Increase Hope Level and Stress Level of Parents With a CMC (NCT05269693) | The Hong Kong Polytechnic University | 2021-12-01 |
| Pediatric Spinal Muscular Atrophy (SMA) China Registry (NCT05042921) | Biogen | 2021-11-18 |
| Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) (NCT05073133) | Novartis Pharmaceuticals | 2021-11-04 |
| A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD) (NCT04729907) | Biogen | 2021-04-19 |
| Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants (NCT04718181) | Hoffmann-La Roche | 2021-02-01 |
| A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND) (NCT04488133) | Biogen | 2021-01-04 |
| Ultrasound-assisted vs Landmark Based Intrathecal Administration of Nusinersen (NCT04674618) | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | 2020-12-14 |
| Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection (NCT04419233) | Biogen | 2020-11-18 |
| Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy (NCT04089566) | Biogen | 2020-03-26 |
| A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (NCT03779334) | Hoffmann-La Roche | 2019-08-07 |
| Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) (NCT04317794) | Biogen | 2019-07-02 |
| A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function (NCT03920865) | Hoffmann-La Roche | 2019-05-16 |
| An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy (NCT03921528) | Scholar Rock, Inc. | 2019-04-22 |
| Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients (NCT03781479) | Catalyst Pharmaceuticals, Inc. | 2019-01-21 |
| Natural History of SMA (NCT05755451) | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | 2018-06-21 |
| Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy (NCT02913482) | Hoffmann-La Roche | 2016-12-23 |
| A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants (NCT02908685) | Hoffmann-La Roche | 2016-10-19 |
| A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA) (NCT02628743) | Hoffmann-La Roche | 2016-01-20 |
| A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH) (NCT02240355) | Hoffmann-La Roche | 2014-11-01 |
| A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis (NCT01910168) | Hoffmann-La Roche | 2013-08-01 |
| A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy (NCT07474311) | Novartis Pharmaceuticals | 2011-11-22 |
| Progressive Strength Training in Spinal Muscular Atrophy (NCT01233817) | University of Utah | 2010-06-01 |
| A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients (NCT00568698) | Stanford University | 2004-01-01 |
| A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients (NCT00568802) | Stanford University | 2004-01-01 |
| International SMA Patient Registry (NCT00466349) | Indiana University | 1986-05-01 |
| An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA) (NCT04256265) | Genentech, Inc. | |