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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, 0 tagged with Qualitative or quantitative defects of beta-sarcoglycan itself.

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Part of

↑ Hereditary neurological disease (5791) ↑ Sarcoglycanopathy (10)

Sub-types of Qualitative or quantitative defects of beta-sarcoglycan

Autosomal recessive limb-girdle muscular dystrophy type 2E 5 trials

Including sub-types (5) Tagged with Qualitative or quantitative defects of beta-sarcoglycan (0)

Trials to join now! 1 Not yet finished but already full! 3 Terminated 1

Experimental gene therapy for rare muscle disease shows early promise but study halted

Disease control Stopped early

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 1/2 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control

Last updated Jun 27, 2026 08:12 UTC