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Qualitative or quantitative defects of alpha-sarcoglycan

MONDO:0016141

Also known as: alpha-sarcoglycanopathy

3 clinical trials for this condition and its sub-types.

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Sub-types

Autosomal recessive limb-girdle muscular dystrophy type 2D (3)

Broader categories

Disease (618) Nervous system disorder (217) Hereditary disease (172) Human disease (14) Hereditary neurological disease (5) Sarcoglycanopathy (3) Disease of genetic or genomic mechanism (2) Disease by body system or component (0) Disease by etiologic mechanism (0) Qualitative or quantitative protein defects in neuromuscular diseases (0)

Trials to join now! 1 Not yet finished but already full! 1 Terminated 1

Experimental gene therapy tested for rare muscular dystrophy

Disease control Terminated

This early-stage trial tested a gene therapy called SRP-9004 in just 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, not effectiveness. The study was terminated early, so results are limited.

Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control

Last updated Jun 26, 2026 13:47 UTC