A blue informational slide about sarcoglycanopathy, a rare disease, with text mentioning current research and clinical trials, and a logo of the Dion Foundation for children with rare diseases, along with a DNA strand graphic on the right.

CLINICAL TRIALS

Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD) (GRASP-01-001)

beta.clinicaltrials.gov

Sponsor:

Virginia Commonwealth University

Status:

Active, not recruiting

ATA-200 Dose-escalation Gene Therapy Trial in Patients with LGMDR5

This is a multicenter Phase 1b assessing the safety and tolerability of 2 doses of ATA-200 for the treatment of LGMDR5.


Atamyo Therapeutics clinicaltrials.gov

Sponsor:

Atamyo Therapeutics

Status:

Recruiting

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