Muscular Atrophy, Spinal
Pipeline by Development Stage
Drug Modality Breakdown
On Market (2)
Approved therapies currently available
Competitive Landscape
6 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Showing 15 of 19 trials with date data
Clinical Trials (19)
Total enrollment: 35,209 patients across 19 trials
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
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)
A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec
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)
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
Pilot Study of Growth Hormon to Treat SMA Typ II and III
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
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
A Study of the Excretion Balance of Radiocarbon and the Pharmacokinetics and Metabolic Profile of TRO19622 (Olesoxime)
Adult Spinal Muscular Atrophy (SMA) China Registry
Newborn Screening for Spinal Muscular Atrophy
Pediatric Spinal Muscular Atrophy (SMA) China Registry
Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection
Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)