Myotonic dystrophy

8 trials 7 recruiting MedDRA PT
8 Registered trials
7 Recruiting
4 Unique sponsors
5 Unique IMPs
8 Commercial trials
Drug classes (ATC)
N Neurology/Psychiatry (ATC N) 1
— ATC code not specified 4
C Cardiology (ATC C) 3
Mechanism of action (INN-stem analysis)
Unknown / small molecule 5 substances
Phase III: 4 Phase I/II: 1 Phase I: 1 Phase I (First in Human): 2

Filter these 8 trials by country, sponsor, date range and more.

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Show: 8 trials

Phase III

4 trials
  • Dyne Therapeutics Inc. Commercial
    HUMANISED IGG1 KAPPA FRAGMENT ANTIBODY TARGETING TFR1 CONJUGATED TO P125 OLIGONUCLEOTIDE
    2025-522957-20-00 7 countries n=72 Orphan
    Pending
  • Lupin Atlantis Holdings SA Commercial
    MEXILETINE HYDROCHLORIDE
    2024-511178-66-00 5 countries 2025 n=146 Orphan
    Recruiting
  • Lupin Atlantis Holdings SA Commercial
    MEXILETINE HYDROCHLORIDE
    2024-511179-13-00 5 countries 2025 n=146 Orphan
    Recruiting
  • Lupin Atlantis Holdings SA Commercial
    MEXILETINE; MEXILETINE HYDROCHLORIDE
    2024-514970-27-00 4 countries 2025 n=20 Orphan
    Recruiting

Phase I/II

1 trial
  • Vertex Pharmaceuticals Inc. Commercial
    VX-670
    2024-517983-47-00 6 countries 2025 n=20
    Recruiting

Phase I

1 trial
  • Arthex Biotech S.L. Commercial
    16-BASE SINGLE STRANDED RNA TARGETING MIR-23B LINKED TO OLEIC ACID
    2023-505363-37-00 4 countries 2024 n=28 Orphan
    Recruiting

Phase I (First in Human)

2 trials
  • Dyne Therapeutics Inc. Commercial
    HUMANISED IGG1 KAPPA FRAGMENT ANTIBODY TARGETING TFR1 CONJUGATED TO P125 OLIGONUCLEOTIDE
    2023-510353-42-00 4 countries 2022 n=103 Orphan
    Recruiting
  • Vertex Pharmaceuticals Inc. Commercial
    VX-670
    2023-506028-10-00 6 countries 2024 n=20
    Recruiting
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
Lupin Atlantis Holdings SA Commercial 3 Phase III 3
Dyne Therapeutics Inc. Commercial 2 Phase III, Phase I (First in Human) 1
Vertex Pharmaceuticals Inc. Commercial 2 Phase I (First in Human), Phase I/II 2
Arthex Biotech S.L. Commercial 1 Phase I 1
New trials by year
2022
1
2024
2
2025
4
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