Qualitative or quantitative defects of FKRP
MONDO:00161568 clinical trials for this condition and its sub-types, 0 tagged with Qualitative or quantitative defects of FKRP itself.
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Browse by category →Sub-types of Qualitative or quantitative defects of FKRP
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Hope for rare muscle disease: new drug enters final testing phase
Disease control OngoingThis study tests a drug called BBP-418 (Ribitol) in 81 people aged 12 to 60 with limb girdle muscular dystrophy type 2I (LGMD2I), a genetic condition that causes progressive muscle weakness. Participants receive either the drug or a placebo for 36 months to see if it slows the di…
Phase 3 • Sponsor: ML Bio Solutions, Inc. • Aim: Disease control
Last updated Sep 11, 2026 00:00 UTC
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Gene therapy trial offers hope for rare muscle disease
Disease control OngoingThis study tests a single dose of a gene therapy called AB-1003 in 10 adults with a rare genetic muscle disease (LGMD2I/R9). The goal is to see if it is safe and can help improve muscle function. Participants must be able to walk or run 10 meters in under 30 seconds.
Phase 1/2 • Sponsor: AskBio Inc • Aim: Disease control
Last updated Aug 28, 2026 00:00 UTC
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Experimental drug targets root cause of rare muscle-wasting disease
Disease control OngoingThis study tests an experimental drug, BBP-418, in people with limb girdle muscular dystrophy type 2I (LGMD2I), a rare genetic condition that causes progressive muscle weakness. The drug aims to fix a molecular defect by helping a faulty enzyme work better. The trial includes bot…
Phase 2 • Sponsor: ML Bio Solutions, Inc. • Aim: Disease control
Last updated Jul 23, 2026 00:00 UTC
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Hope for muscle strength: experimental drug tested for Long-Term use in rare muscular dystrophy
Disease control OngoingThis study tests the long-term safety and effectiveness of an experimental drug called BBP-418 (ribitol) in people with limb-girdle muscular dystrophy type 2I/R9, a rare genetic muscle-weakening disease. Participants who completed a previous study will take BBP-418 orally twice d…
Phase 3 • Sponsor: ML Bio Solutions, Inc. • Aim: Disease control
Last updated Jul 04, 2026 00:00 UTC
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Experimental gene therapy targets rare muscle disease
Disease control OngoingThis early-stage trial tests a gene therapy called ATA-100 for people with LGMDR9, a rare genetic muscle disease that causes progressive weakness. Six adults receive a single intravenous infusion of the therapy, which delivers a working copy of the FKRP gene. The main goal is to …
Phase 1 • Sponsor: Atamyo Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:54 UTC
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Researchers hunt for muscle clues in rare dystrophy
Knowledge-focused OngoingThis pilot study looks at biomarkers in the blood of people with fragile sarcolemmal muscular dystrophy, a condition that makes muscle membranes weak. Researchers will collect blood samples at rest and after exercise during four 5-day hospital stays. The goal is to better underst…
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated Sep 18, 2026 00:00 UTC