Qualitative or quantitative defects of gamma-sarcoglycan
MONDO:0016143Also known as: gamma-sarcoglycanopathy
4 clinical trials for this condition and its sub-types.
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Broader categories
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Gene therapy hope for kids with rare muscle-wasting disease
Disease control OngoingThis early-stage trial tests a single intravenous dose of a gene therapy (ATA-200) in 4 children aged 6-12 with limb-girdle muscular dystrophy type 2C/R5 (LGMD2C), a rare genetic muscle-weakening condition. The goal is to see if the treatment is safe and tolerable by delivering a…
Phase: PHASE1 • Sponsor: Atamyo Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Scientists watch LGMD progress in 205 patients over years
Knowledge-focused OngoingThis study follows 205 people with four types of limb-girdle muscular dystrophy (LGMD) to understand how the disease changes over time. Participants will have their muscle strength, movement, and breathing tested regularly for up to 5 years. No treatment is given; the goal is to …
Sponsor: Sarepta Therapeutics, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:56 UTC
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New study aims to map rare muscle disease progression
Knowledge-focused TerminatedThis study was designed to track the natural course of gamma-sarcoglycanopathy (LGMDR5), a rare muscle-weakening disease, over two years. Researchers planned to measure changes in muscle strength, walking ability, and daily function in patients aged 6 to 35. The goal was to bette…
Sponsor: Atamyo Therapeutics • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:54 UTC