MUSCULAR DISEASES
Clinical trials for MUSCULAR DISEASES explained in plain language.
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New hope for boys with rare muscle disease as experimental drug opens for access
Disease control AVAILABLEThis program provides access to an experimental drug called AOC 1044 for people with Duchenne muscular dystrophy who have specific genetic mutations. The treatment aims to help muscles produce a more functional protein by 'skipping' a problematic section of genetic code. Eligible…
Matched conditions: MUSCULAR DISEASES
Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Apr 03, 2026 22:56 UTC
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Scientists test revolutionary immune cell therapy for debilitating muscle disease
Disease control Recruiting nowThis is an early-stage safety study testing a new type of treatment called anito-cel for adults with generalized myasthenia gravis, a chronic autoimmune disease that causes severe muscle weakness. The therapy involves collecting a patient's own immune cells, genetically modifying…
Matched conditions: MUSCULAR DISEASES
Phase: PHASE1 • Sponsor: Arcellx, Inc. • Aim: Disease control
Last updated Mar 23, 2026 15:17 UTC