Duchenne muscular dystrophy

25 trials 7 recruiting MedDRA PT
25 Registered trials
7 Recruiting
16 Unique sponsors
22 Unique IMPs
22 Commercial trials
Drug classes (ATC)
— ATC code not specified 21
N Neurology/Psychiatry (ATC N) 2
H Endocrinology (ATC H) 1
C Cardiology (ATC C) 1
B Haematology (ATC B) 1
L Oncology (ATC L) 1
Mechanism of action (INN-stem analysis)
Unknown / small molecule 17 substances
HDAC inhibitor 1 substance
Antisense oligonucleotide 1 substance
Beta-blocker 1 substance
Complement inhibitor (C5) 1 substance
mTOR inhibitor 1 substance
Phase III: 10 Phase II: 4 Phase I/II: 1 Phase IV: 1 Phase I (Other): 3 Phase I (First in Human): 7

Filter these 25 trials by country, sponsor, date range and more.

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Show: 25 trials

Phase III

10 trials
  • Avidity Biosciences Inc. Commercial
    DELPACIBART ZOTADIRSEN
    2025-523087-20-00 7 countries n=42 Orphan
    Pending
  • Italfarmaco S.p.A. Commercial
    GIVINOSTAT
    2023-504520-26-00 6 countries 2017 n=120 Orphan
    Recruiting
  • Ns Pharma Inc. Commercial
    VILTOLARSEN
    2023-507146-91-00 6 countries 2021 n=21 Orphan
    Ended
  • Pfizer Inc. Commercial
    FORDADISTROGENE MOVAPARVOVEC
    2023-508510-42-00 5 countries 2025 n=48 Orphan
    Ended
  • Sarepta Therapeutics Inc. Commercial
    DELANDISTROGENE MOXEPARVOVEC
    2023-505043-39-00 4 countries 2024 n=74 Orphan
    Recruiting
  • Sarepta Therapeutics Inc. Commercial
    ETEPLIRSEN
    2024-511492-15-00 13 countries 2021 n=37 Orphan
    Ended
  • Sarepta Therapeutics Inc. Commercial
    DELANDISTROGENE MOXEPARVOVEC
    2024-512626-28-00 6 countries 2024 n=95 Orphan
    Halted
  • Sarepta Therapeutics Inc. Commercial
    CASIMERSEN; GOLODIRSEN
    2024-514698-23-00 9 countries 2017 n=81
    Ended
  • Solid Biosciences Inc. Commercial
    SGT-003
    2025-522949-22-00 6 countries n=62
    Pending
  • Medical University Of Gdansk Academic
    METOPROLOL TARTRATE
    2024-515057-19-00 1 country 2021 n=144
    Ended

Phase II

4 trials
  • Antisense Therapeutics Limited Commercial
    2'-O-(2-METHOXYETHYL) PHOSPHOROTHIOATE ANTISENSE OLIGONUCLEOTIDE TARGETING CD49D RNA
    2024-512265-13-00 1 country 2023 n=1 Orphan
    Ended
  • Biomarin Pharmaceutical Inc. Commercial
    BMN 351
    2024-511656-41-00 3 countries n=5
    Pending
  • F. Hoffmann-La Roche AG Commercial
    DELANDISTROGENE MOXEPARVOVEC
    2023-509901-57-00 5 countries 2023 n=11 Orphan
    Halted
  • F. Hoffmann-La Roche AG Commercial
    IMP not specified
    2024-512383-65-00 4 countries 2024 n=28
    Ended

Phase I/II

1 trial
  • Entrada Therapeutics Inc. Commercial
    ENTR-601-44
    2024-517584-23-00 3 countries 2025 n=9
    Recruiting

Phase IV

1 trial
  • Santhera Pharmaceuticals (Schweiz) AG Commercial
    VAMOROLONE
    2024-512828-12-00 6 countries 2024 n=26 Orphan
    Ended

Phase I (Other)

3 trials
  • Santhera Pharmaceuticals (Schweiz) AG Commercial
    IMP not specified
    2024-512101-60-00 1 country 2024 n=30
    Ended
  • Santhera Pharmaceuticals (Schweiz) AG Commercial
    IMP not specified
    2024-513845-36-00 1 country 2024 n=18
    Ended
  • Santhera Pharmaceuticals (Schweiz) AG Academic
    IMP not specified
    2024-512101-60-00 1 country 2024 n=30
    Ended

Phase I (First in Human)

7 trials
  • Biomarin Pharmaceutical Inc. Commercial
    BMN 351
    2023-506737-30-00 3 countries 2024 n=6
    Ended
  • Dyne Therapeutics Inc. Commercial
    FRAGMENT ANTIBODY TARGETING HUMAN TFR1 CONJUGATED TO PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER
    2023-510351-31-00 4 countries 2023 n=16
    Ended
  • Dystrogen Therapeutics Technology Polska Sp. z o.o. Commercial
    EX VIVO FUSED NORMAL ALLOGENEIC HUMAN MYOBLAST WITH AUTOLOGOUS HUMAN MYOBLAST DERIVED FROM DUCHENNE MUSCULAR DYSTROPHY AFFECTED DONOR
    2024-519004-27-00 1 country n=20 Orphan
    Pending
  • Entrada Therapeutics Inc. Commercial
    ENTR-601-45
    2024-517499-39-00 4 countries 2025 n=14
    Recruiting
  • Solid Biosciences Inc. Commercial
    SGT-003
    2024-514501-57-00 1 country 2025 n=8
    Recruiting
  • Genethon Academic
    ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE 8 CONTAINING THE HUMAN MD1 GENE; ECULIZUMAB; SIROLIMUS; SODIUM LACTATE SOLUTION, POTASSIUM CHLORIDE, SODIUM CHLORIDE, CALCIUM CHLORIDE DIHYDRATE
    2023-505187-11-00 3 countries 2021 n=40 Orphan
    Recruiting
  • Sqy Therapeutics Academic
    SQY51
    2022-500703-49-01 1 country 2025 n=12
    Recruiting
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
Santhera Pharmaceuticals (Schweiz) AG Commercial 4 Phase I (Other), Phase IV 0
Sarepta Therapeutics Inc. Commercial 4 Phase III 1
Biomarin Pharmaceutical Inc. Commercial 2 Phase II, Phase I (First in Human) 0
F. Hoffmann-La Roche AG Commercial 2 Phase II 0
Solid Biosciences Inc. Commercial 2 Phase III, Phase I (First in Human) 1
Entrada Therapeutics Inc. Commercial 2 Phase I (First in Human), Phase I/II 2
Antisense Therapeutics Limited Commercial 1 Phase II 0
Avidity Biosciences Inc. Commercial 1 Phase III 0
Italfarmaco S.p.A. Commercial 1 Phase III 1
Ns Pharma Inc. Commercial 1 Phase III 0
Pfizer Inc. Commercial 1 Phase III 0
Dyne Therapeutics Inc. Commercial 1 Phase I (First in Human) 0
Dystrogen Therapeutics Technology Polska Sp. z o.o. Commercial 1 Phase I (First in Human) 0
Medical University Of Gdansk Academic 1 Phase III 0
Genethon Academic 1 Phase I (First in Human) 1
Sqy Therapeutics Academic 1 Phase I (First in Human) 1
New trials by year
2021
4
2023
3
2024
8
2025
5
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