Clinical trials: Limb Girdle Muscular Dystrophy
Limb-Girdle Muscular Dystrophy, Type 2D
A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)
This is an open-label, dose escalation gene transfer therapy study evaluating the safety of SRP-9004 (patidistrogene bexoparvovec) via isolated limb infusion (ILI) administration in approximately 6 participants with LGMD2D.
- Trial name
- NCT01976091
- Testing
- SRP-9004
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Facioscapulohumeral Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
ATYR1940-C-006 is a multi-national, multicenter study being conducted at centers in the United States (US) and Europe who participated in Study ATYR1940-C-003 (Stage 1 only) or Study ATYR1940-C-004 (that is, the parent studies).
- Trial name
- FSHD (NCT02836418)
- Testing
- ATYR1940
- Where
- 5 sites in United States, Denmark and 1 more
- Volunteers
- 8 people
- Sponsor
- aTyr Pharma, Inc.