Hope for rare muscle disease: new drug enters final testing phase
NCT ID NCT05775848
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This 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 disease and is safe. The main goal is to measure changes in muscle function and track any side effects.
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Charité Universitätsmedizin Berlin and Max Delbrück Center
Berlin, Germany
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Children's Hospital of the Kings Daughters
Norfolk, Virginia, 23455, United States
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Great Ormond Street Hospital for Children
London, United Kingdom
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IRCCS Ca' Granda Ospedale
Milan, Italy
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International Centre for Life
Newcastle upon Tyne, United Kingdom
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Leiden University Medical Center
Leiden, Netherlands
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Oregon Health & Science University (OHSU) - Neurology Clinic - South Waterfront
Portland, Oregon, 97239, United States
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Rigshospitalet, Neuromuscular Clinic and Research Unit
Copenhagen, Denmark
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Royal Brisbane and Women's Hospital,
Brisbane, Queensland, Australia
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Universitetssykehuset Nord-Norge, Department of Neurology
Tromsø, Norway
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University of California Irvine
Irvine, California, 92697, United States
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University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045, United States
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University of Florida
Gainsville, Florida, 32610, United States
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University of Iowa
Iowa City, Iowa, 52242, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Minnesota, Twin Cities
Minneapolis, Minnesota, 55455, United States
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University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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