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Qualitative or quantitative defects of beta-sarcoglycan

MONDO:0016142

Also known as: beta-sarcoglycanopathy

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

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Sub-types

Autosomal recessive limb-girdle muscular dystrophy type 2E (5)

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! 3 Terminated 1
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  • Experimental gene therapy for rare muscle disease shows early promise but study halted

    Disease control Terminated

    This study tested a gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E (LGMD2E), a rare genetic disease that causes muscle weakness. The treatment aimed to deliver a working gene to muscle cells to help them produce a missing protein. Only 6 peopl…

    Phase: PHASE1, PHASE2 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control

    Last updated Jun 27, 2026 08:12 UTC

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