| Not Yet Recruiting | A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscula NCT07444450 | Biogen | Phase 3 |
| Recruiting | A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Ap NCT07221669 | Biogen | Phase 3 |
| Completed | A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy NCT07403214 | Novartis Pharmaceuticals | — |
| Recruiting | A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Ad NCT06555419 | Biogen | Phase 1 |
| Completed | A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Ther NCT07378943 | Novartis Pharmaceuticals | — |
| Recruiting | A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Mu NCT05861999 | Hoffmann-La Roche | Phase 4 |
| Recruiting | A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatri NCT05861986 | Hoffmann-La Roche | Phase 4 |
| Recruiting | A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy NCT05808764 | Hoffmann-La Roche | Phase 2 |
| Completed | Two Different Treatment Modalities in Patients With Spinal Muscular Atrophy NCT06178653 | Istanbul Medipol University Hospital | N/A |
| Recruiting | A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it NCT05789758 | Biogen | — |
| Active Not Recruiting | Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab NCT05626855 | Scholar Rock, Inc. | Phase 3 |
| Active Not Recruiting | Adult Spinal Muscular Atrophy (SMA) China Registry NCT05618379 | Biogen | — |
| Active Not Recruiting | A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy NCT05575011 | Biogen | Phase 1 |
| Completed | Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinerse NCT05156320 | Scholar Rock, Inc. | Phase 3 |
| Completed | Newborn Screening for Spinal Muscular Atrophy NCT05481164 | University of Oxford | — |
| Completed | Procedural Sedation for Pediatric Patients With Spinal Muscular Atrophy NCT05291962 | Ankara City Hospital Bilkent | — |
| Completed | A BHI to Increase Hope Level and Stress Level of Parents With a CMC NCT05269693 | The Hong Kong Polytechnic University | N/A |
| Active Not Recruiting | Pediatric Spinal Muscular Atrophy (SMA) China Registry NCT05042921 | Biogen | — |
| Completed | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) NCT05073133 | Novartis Pharmaceuticals | Phase 4 |
| Active Not Recruiting | A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Par NCT04729907 | Biogen | Phase 3 |
| Completed | Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants NCT04718181 | Hoffmann-La Roche | Phase 1 |
| Completed | A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular NCT04488133 | Biogen | Phase 4 |
| Completed | Ultrasound-assisted vs Landmark Based Intrathecal Administration of Nusinersen NCT04674618 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | N/A |
| Completed | Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection NCT04419233 | Biogen | — |
| Completed | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy NCT04089566 | Biogen | Phase 3 |
| Active Not Recruiting | A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy NCT03779334 | Hoffmann-La Roche | Phase 2 |
| Active Not Recruiting | Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) NCT04317794 | Biogen | — |
| Completed | A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of NCT03920865 | Hoffmann-La Roche | Phase 1 |
| Completed | An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy NCT03921528 | Scholar Rock, Inc. | Phase 2 |
| Completed | Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients NCT03781479 | Catalyst Pharmaceuticals, Inc. | Phase 2 |
| Recruiting | Natural History of SMA NCT05755451 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | — |
| Completed | Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Mu NCT02913482 | Hoffmann-La Roche | Phase 2 |
| Completed | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam NCT02908685 | Hoffmann-La Roche | Phase 2 |
| Completed | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Mus NCT02628743 | Hoffmann-La Roche | Phase 2 |
| Terminated | A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH) NCT02240355 | Hoffmann-La Roche | Phase 1 |
| Completed | A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis NCT01910168 | Hoffmann-La Roche | — |
| Completed | A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy NCT07474311 | Novartis Pharmaceuticals | — |
| Completed | Progressive Strength Training in Spinal Muscular Atrophy NCT01233817 | University of Utah | N/A |
| Completed | Pilot Study of Growth Hormon to Treat SMA Typ II and III NCT00533221 | University Hospital Freiburg | Phase 2 |
| Completed | A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients NCT00568802 | Stanford University | Phase 1 / Phase 2 |
| Completed | A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients NCT00568698 | Stanford University | Phase 1 / Phase 2 |
| Completed | International SMA Patient Registry NCT00466349 | Indiana University School of Medicine | — |
| Approved For Marketing | An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA) NCT04256265 | Genentech, Inc. | — |