Landscape → Rare Disease

Spinal Muscular Atrophy Clinical Trial Landscape

Data updated: May 20, 2026

61 clinical trials tracked since 2008

Spinal Muscular Atrophy is being studied across 61 clinical trials registered since 2008, with 24 programs currently active. The competitive pipeline includes 10 active Phase 3 trials, 12 active Phase 2 trials, and 8 active Phase 1 trials. Top industry sponsors include GeneCradle Inc, Biogen, Roche.

24 active

61 total (since 2008)

Phase 1 Active

20 total

Phase 2 Active

30 total

Phase 3 Active

22 total

Phase 4 Active

4 total

Top Industry Sponsors

Sorted by active Active Done Failed

GeneCradle Inc 4 0 0

Biogen 2 8 2

Roche 2 4 0

Novartis 2 4 0

Scholar Rock, Inc. 2 2 0

+ 9 more sponsors — see full table below

Featured Phase 3 Trials

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

NCT05115110 ACTIVE NOT RECRUITING

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=259

NCT05331937 RECRUITING

TMS for Exposure Therapy Resistant OCD

Amsterdam UMC, location VUmc n=250

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 RECRUITING

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

Novartis Pharmaceuticals n=175

+ 17 more Phase 3 trials — see full database below

Spinal Muscular Atrophy — Trials Started by Year (last 10 years)

2016

2017

2018

2019

2020

2021

2022

2023

2024

2025

2026

TheraRadar.com

Full Trial Database

Complete sponsor table, every trial across all phases, plus competitive analytics. Sortable, filterable, exportable for Pro users.

Top Sponsors (all-time)

GeneCradle Inc

Biogen

Roche

Novartis

Scholar Rock, Inc.

Lantu Biopharma

Novartis Gene Therapies

Lanyue Biotech (Hangzhou) Co., Ltd.

argenx

NMD Pharma A/S

Active Trials by Phase

Phase 1

Phase 2

Phase 3

Phase 4

Enrollment Size Distribution

1-50

51-200

201-500

501-1k

1k+

All Industry Sponsors

Sorted by active Active Done Failed

GeneCradle Inc 4 0 0

Biogen 2 8 2

Roche 2 4 0

Novartis 2 4 0

Scholar Rock, Inc. 2 2 0

Lantu Biopharma 2 0 0

Novartis Gene Therapies 1 5 1

Lanyue Biotech (Hangzhou) Co., Ltd. 1 0 0

argenx 1 0 0

NMD Pharma A/S 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

NCT07444476 RECRUITING

Biogen n=90

NCT05115110 ACTIVE NOT RECRUITING

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=259

NCT05331937 RECRUITING

TMS for Exposure Therapy Resistant OCD

Amsterdam UMC, location VUmc n=250

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 RECRUITING

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

Novartis Pharmaceuticals n=175

NCT07265232 RECRUITING

Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).

Lantu Biopharma n=15

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

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

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

NCT07047144 RECRUITING

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

Scholar Rock, Inc.

NCT07554924 NOT YET RECRUITING

A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I

Lanyue Biotech (Hangzhou) Co., Ltd.

NCT07287982 RECRUITING

A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy

argenx

NCT05794139 ACTIVE NOT RECRUITING

Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

NMD Pharma A/S

NCT07070999 RECRUITING

Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

Gemma Biotherapeutics

NCT06288230 RECRUITING

An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy

Lantu Biopharma

NCT05824169 RECRUITING

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

GeneCradle Inc

NCT06421831 RECRUITING

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

GeneCradle Inc

NCT05901987 ACTIVE NOT RECRUITING

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

GeneCradle Inc

NCT05614531 ENROLLING BY INVITATION

Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

Guangzhou Jiayin Biotech Ltd

NCT05747261 RECRUITING

Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)

Biocad

NCT02386553 COMPLETED

A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Biogen

NCT03032172 COMPLETED

A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

Hoffmann-La Roche

NCT02268552 COMPLETED

An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)

Novartis Pharmaceuticals

NCT03921528 COMPLETED

An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy

Scholar Rock, Inc.

NCT03819660 TERMINATED

Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3

Catalyst Pharmaceuticals, Inc.

NCT02227823 COMPLETED

Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3

Centre Hospitalier Régional de la Citadelle

NCT01703988 COMPLETED

An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy

Biogen

NCT01839656 COMPLETED

A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)

Biogen

NCT02462759 TERMINATED

A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).

Biogen

NCT02876094 TERMINATED

Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy

Hugh McMillan

NCT02644668 COMPLETED

A Study of CK-2127107 in Patients With Spinal Muscular Atrophy

Cytokinetics

NCT02941328 COMPLETED

SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4

UMC Utrecht

NCT01302600 COMPLETED

Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.

Hoffmann-La Roche

NCT01028833 COMPLETED

Effects of Power Mobility on Young Children With Severe Motor Impairments

University of Oklahoma

NCT00661453 COMPLETED

CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I

University of Utah

NCT00439218 TERMINATED

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I

Westat

NCT00439569 TERMINATED

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III

Westat

Full Spinal Muscular Atrophy Pipeline

Every sponsor, every trial across Phase 1–4. Sortable, filterable, exportable.

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Data: ClinicalTrials.gov · Trials registered 2008 onwards · Industry sponsors only