Clinical trials: Limb-Girdle Muscular Dystrophy, Type 2D

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)

• Completed
• Age: 7 years and older
• Phase 1/2
• Results posted

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.