LIMB GIRDLE MUSCULAR DYSTROPHY
Clinical trials for LIMB GIRDLE MUSCULAR DYSTROPHY explained in plain language.
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One-Time gene therapy aims to halt rare muscle disease
Disease control OngoingThis study tests a single dose of SRP-9003 gene therapy in 17 people with limb girdle muscular dystrophy 2E/R4, a genetic muscle-weakening disease. The goal is to restore a missing protein in muscle cells and improve muscle function. Both walkers and non-walkers can join, and the…
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE3 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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Experimental gene therapy targets rare muscle disease in first human test
Disease control OngoingThis early-phase trial tests a single-dose gene therapy called SRP-9003 in 6 people with limb girdle muscular dystrophy type 2E/R4, a rare genetic muscle-weakening disease. The main goals are to check safety and see if the therapy can produce the missing beta-sarcoglycan protein …
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC
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Paving the way: new study aims to sharpen tools for LGMD R1 trials
Knowledge-focused OngoingThis 24-month observational study follows 100 people aged 12–50 with Limb Girdle Muscular Dystrophy type R1 (LGMD R1). Researchers will test whether a motor function scale called NSAD and muscle fat measurements from MRI can reliably track disease progression. The goal is to vali…
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Sponsor: Virginia Commonwealth University • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:07 UTC
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New study tracks muscle decline in rare muscular dystrophy
Knowledge-focused OngoingThis study follows 25 people with limb-girdle muscular dystrophy type 2A (LGMD2A), a rare genetic disease that causes progressive muscle weakness. Researchers will measure how muscle strength changes over time and how it affects quality of life. The goal is to better understand t…
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 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 …
Matched conditions: LIMB GIRDLE MUSCULAR DYSTROPHY
Sponsor: Sarepta Therapeutics, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:56 UTC