CLINICAL TRIALS
A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, and LGMD2C/R5, ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
Sponsor:
Sarepta Therapeutics, Inc.
Status:
Recruiting
Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD) (GRASP-01-001)
Sponsor:
Virginia Commonwealth University
Status:
Recruiting
Orphan Drug Designation awarded from European EMA for ATA-200
This product was licensed to Atamyo for clinical development and a clinical trial application is expected to be filed shortly.
Atamyo Therapeutics genethon.com
Sponsor:
Atamyo Therapeutics
Status:
Preparing
RESEARCH & DEVELOPMENT
gene therapy
An AAV-SGCG Dose-Response Study in a γ-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress READ MORE
A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C READ MORE
Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model READ MORE
EXON SKIPPING
A gene-edited mouse model of limb-girdle muscular dystrophy 2C for testing exon skipping. READ MORE