Data updated: Nov 12, 2026
Spinal Muscular Atrophy Clinical Trial Landscape
Spinal Muscular Atrophy is being studied across 62 clinical trials registered since 2008, with 21 programs currently active. The competitive pipeline includes 9 active Phase 3 trials, 10 active Phase 2 trials, and 0 active Phase 1 trials.
Top industry sponsors include GeneCradle Inc, Biogen, Novartis.
Trial activity
21 active / 62 total since 2008
Active by phase 9 Ph3 / 23 10 Ph2 / 28 0 Ph1 / 7 2 Ph4 / 4
Sponsor activity
Who is running trials now — green active, blue completed, red failed/terminated.
Sorted by active Active Done Failed
GeneCradle Inc 4 0 0
Biogen 2 8 2
Novartis 2 4 0
Scholar Rock, Inc. 2 2 0
Novartis Gene Therapies 1 5 1
Lantu Biopharma 1 0 1
Lanyue Biotech (Hangzhou) Co., Ltd. 1 0 0
argenx 1 0 0
Gemma Biotherapeutics 1 0 0
Biohaven Pharmaceuticals, Inc. 1 0 0
Guangzhou Jiayin Biotech Ltd 1 0 0
Biocad 1 0 0
Roche 0 6 0
How the field has grown
New-trial starts peaked in 2022 (9 registered); 2025 saw 4. The right-hand chart shows median Phase 3 enrollment by start year — the number in parentheses is that year's Phase 3 trial count (18 in total), so single-trial years (and years with no Phase 3 starts) are obvious. Both are by trial start date; the current year is partial.
New trials started by year
2016 2
2017 3
2018 2
2019 5
2020 1
2021 1
2022 9
2023 7
2024 2
2025 4
2026 5
TheraRadar.com
Median Phase 3 enrollment by start year
2016 (0) 0
2017 (1) 22
2018 (2) 32
2019 (1) 2
2020 (1) 85
2021 (1) 24
2022 (7) 126
2023 (2) 133
2024 (0) 0
2025 (1) 50
2026 (2) 46
TheraRadar.com
Full trial pipeline
Every active and completed trial across Phase 1–4, with enrollment analytics. Sortable, filterable, exportable with Pro.
NCT07444476 RECRUITING
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam
Biogen n=90
NCT05337553 ACTIVE NOT RECRUITING
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
Biohaven Pharmaceuticals, Inc. n=269
NCT05335876 ACTIVE NOT RECRUITING
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
Novartis Pharmaceuticals n=20
NCT06971094 RECRUITING
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
GeneCradle Inc n=50
NCT05626855 ACTIVE NOT RECRUITING
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
Scholar Rock, Inc. n=238
NCT05067790 ACTIVE NOT RECRUITING
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
Biogen n=45
NCT04042025 ACTIVE NOT RECRUITING
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Novartis Gene Therapies n=85
NCT07846696 NOT YET RECRUITING
An Open Label Individual Patient (OLIP)-02 Study of Nusinersen (BIIB058)
London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's n=1
NCT05115110 COMPLETED
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Hoffmann-La Roche n=97
NCT03306277 COMPLETED
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Novartis Gene Therapies n=22
NCT03837184 COMPLETED
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
Novartis Gene Therapies n=2
NCT03505099 COMPLETED
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
Novartis Gene Therapies n=30
NCT03461289 COMPLETED
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Novartis Gene Therapies n=33
NCT05156320 COMPLETED
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
Scholar Rock, Inc. n=188
NCT05089656 COMPLETED
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
Novartis Pharmaceuticals n=126
NCT05386680 COMPLETED
Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
Novartis Pharmaceuticals n=27
NCT07265232 WITHDRAWN
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
Lantu Biopharma
NCT02594124 COMPLETED
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
Biogen n=292
NCT04851873 COMPLETED
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
Novartis Pharmaceuticals n=24
NCT01645787 COMPLETED
Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients
Columbia University n=11
NCT02193074 TERMINATED
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
Biogen n=122
NCT02292537 COMPLETED
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
Biogen n=126
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In-depth analysis connected to Spinal Muscular Atrophy.
Data: ClinicalTrials.gov · Trials registered 2008 onwards · Industry sponsors only