Spinal muscular atrophy

10 trials 4 recruiting MedDRA PT
10 Registered trials
4 Recruiting
6 Unique sponsors
5 Unique IMPs
9 Commercial trials
Drug classes (ATC)
— ATC code not specified 4
M Musculoskeletal (ATC M) 6
Mechanism of action (INN-stem analysis)
Unknown / small molecule 4 substances
Monoclonal antibody 1 substance
Phase III: 3 Phase II: 4 Phase IV: 2 Phase I: 1

Filter these 10 trials by country, sponsor, date range and more.

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Show: 10 trials

Phase III

3 trials
  • Biohaven Therapeutics Ltd. Commercial
    TALDEFGROBEP ALFA
    2024-511852-42-00 7 countries 2023 n=104 Orphan
    Ended
  • Novartis Pharma AG Commercial
    ONASEMNOGENE ABEPARVOVEC
    2024-511707-42-00 6 countries 2023 n=26 Orphan
    Ended
  • UZ Leuven Academic
    RISDIPLAM
    2022-501760-17-00 1 country 2023 n=20 Orphan
    Ended

Phase II

4 trials
  • F. Hoffmann-La Roche AG Commercial
    RISDIPLAM
    2023-505602-42-00 6 countries 2024 n=8
    Recruiting
  • F. Hoffmann-La Roche AG Commercial
    RISDIPLAM
    2023-506009-20-00 2 countries 2019 n=6 Orphan
    Ended
  • F. Hoffmann-La Roche AG Commercial
    RISDIPLAM
    2023-506739-14-00 5 countries 2017 n=110 Orphan
    Ended
  • Scholar Rock Inc. Commercial
    APITEGROMAB
    2024-520130-29-00 5 countries 2025 n=30 Orphan
    Recruiting

Phase IV

2 trials
  • F. Hoffmann-La Roche AG Commercial
    RISDIPLAM
    2023-504508-26-00 2 countries 2024 n=15
    Recruiting
  • F. Hoffmann-La Roche AG Commercial
    RISDIPLAM
    2023-505161-81-00 2 countries 2024 n=15
    Recruiting

Phase I

1 trial
  • Biogen Idec Research Limited Commercial
    BIIB115
    2023-505643-39-00 6 countries 2023 n=56
    Ended
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
F. Hoffmann-La Roche AG Commercial 5 Phase II, Phase IV 3
Biogen Idec Research Limited Commercial 1 Phase I 0
Scholar Rock Inc. Commercial 1 Phase II 1
Biohaven Therapeutics Ltd. Commercial 1 Phase III 0
Novartis Pharma AG Commercial 1 Phase III 0
UZ Leuven Academic 1 Phase III 0
New trials by year
2019
1
2023
4
2024
3
2025
1
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