New drug shows promise for muscle stiffness in rare disease
NCT ID NCT06204809
First seen Nov 01, 2025 · Last updated May 11, 2026 · Updated 22 times
Summary
This early-stage study tested a single dose of a new drug called PGN-EDODM1 in 24 adults with myotonic dystrophy type 1 (DM1), a condition that causes muscle weakness and stiffness. The main goal was to check if the drug is safe and how the body processes it. Researchers monitored participants for side effects and measured drug levels in the blood over 16 weeks.
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This is a summary of
the original study
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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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Contacts and locations
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Locations
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CIUSSS du Saguenay-Lac-Saint-Jean
Chicoutimi, Quebec, Canada
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Ottawa Hospital Research Institute (OHRI)
Ottawa, Ontario, Canada
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Rare Disease Research
Atlanta, Georgia, 30329, United States
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Salford Royal Hospital
Salford, United Kingdom
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Stanford University
Palo Alto, California, 94304, United States
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UCI Center for Clinical Research
Irvine, California, 92697, United States
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University College London Hospital
London, UK, NW1 2PG, United Kingdom
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University of Calgary
Calgary, Alberta, T3M 1M4, Canada
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University of Kansas Medical Center
Fairway, Kansas, 66205, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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Virginia Commonwealth University
Richmond, Virginia, 23298, United States
Conditions
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