Clinical Trials
For SARCOGLICANOPATHIES
LGMD2E-R4
2024
Sarepta Therapeutics launches a multicenter clinical trial (ID SRP-9003-301) of gene therapy for LGMD2E-R4 in the United States, United Kingdom, Italy, Belgium, Germany, and Spain.
2022
Sarepta Therapeutics announced a second clinical trial, Voyagene (ID SRP-9003-102), for LGMD2E-R4 in the United States, in Ohio and Tennessee.
2018
First clinical trial (ID SRP-9003-101) of gene therapy by Myonexus-Sarepta Therapeutics for LGMD2E-R4 in the United States, Ohio.
LGMD2D-R3
2025
Clinical trial Discovery (ID SRP-9004-102) of gene therapy by Sarepta Therapeutics for LGMD2D-R3 in the United States, in Ohio and Virginia. A patient died in 2025. The study was terminated.
2015
Clinical trial of gene therapy (ID 9004-101) by Sarepta Therapeutics for LGMD2D-R3 in the United States, at Nationwide Children's Hospital in Columbus, Ohio.
2008
First clinical trial of gene therapy in the United States, developed by Dr. Jerry Mendell, at Nationwide Children's Hospital in Columbus, Ohio, US.
LGMD2C-R5
2025
Clinical trial of gene therapy (ATA-003-GSAR) by Atamyo Therapeutics for LGMD2C-R5, in Florida, US.
2006
Clinical trial of gene therapy (GTG001.06) by Genethon in France for LGMD2C-R5.