MYOTONIC DYSTROPHY
Clinical trials for MYOTONIC DYSTROPHY explained in plain language.
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First gene therapy trial targets root cause of debilitating muscle disease
Disease control Recruiting nowThis study is testing a new, one-time gene therapy called SAR446268 for people aged 10 to 50 with myotonic dystrophy type 1 (DM1). The main goals are to see if the treatment is safe and if it can reduce the harmful genetic material that causes the disease, potentially improving m…
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE1, PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated Feb 23, 2026 14:54 UTC
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Breakthrough blood test could replace risky needle for prenatal diagnosis
Diagnosis Recruiting nowThis study is evaluating a new, non-invasive blood test to diagnose serious inherited genetic disorders in unborn babies. The test analyzes tiny bits of the baby's DNA found in the mother's blood, aiming to provide a safe and accurate diagnosis as early as 9 weeks into pregnancy.…
Matched conditions: MYOTONIC DYSTROPHY
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Diagnosis
Last updated Mar 30, 2026 14:30 UTC
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New hope for easing debilitating muscle lock in rare disease
Symptom relief Recruiting nowThis study is testing whether a daily, extended-release version of the drug mexiletine can safely reduce muscle stiffness (myotonia) in people with myotonic dystrophy types 1 and 2. About 176 participants will take either the real drug or a placebo for 26 weeks to see if it helps…
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE3 • Sponsor: Lupin Ltd. • Aim: Symptom relief
Last updated Mar 27, 2026 12:38 UTC
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Could a simple urine test replace painful muscle biopsies?
Knowledge-focused Recruiting nowThis study aims to find less invasive ways to measure muscular dystrophy disease activity. Researchers will test whether urine and blood samples, along with painless muscle scans, can track the disease as effectively as current muscle biopsies. The goal is to develop better tools…
Matched conditions: MYOTONIC DYSTROPHY
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:42 UTC
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National registry opens doors for rare muscle disease research
Knowledge-focused Recruiting nowThis study creates a national registry to connect people with myotonic dystrophy or facioscapulohumeral muscular dystrophy (FSHD) with researchers. It aims to better understand these rare muscle diseases by tracking symptoms over time and helping match interested participants to …
Matched conditions: MYOTONIC DYSTROPHY
Sponsor: University of Rochester • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:34 UTC
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New scan could replace painful biopsies for muscle disease
Knowledge-focused Recruiting nowThis study aims to see if detailed MRI scans can accurately measure muscle health in people with myotonic dystrophy, a progressive muscle-wasting disease. Researchers will compare scan results with physical strength tests and other health measures in 75 participants. The goal is …
Matched conditions: MYOTONIC DYSTROPHY
Sponsor: Wake Forest University Health Sciences • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:29 UTC
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Blood and urine tests could replace painful muscle biopsies for tracking rare disease
Knowledge-focused Recruiting nowThis study aims to find easier ways to measure myotonic dystrophy activity and severity without needing painful muscle biopsies. Researchers will collect blood and urine samples from about 215 people with or without the condition to look for specific RNA markers. The goal is to s…
Matched conditions: MYOTONIC DYSTROPHY
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated Mar 18, 2026 18:25 UTC
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New portable scanner could track walking changes in muscle diseases
Knowledge-focused Recruiting nowThis study is testing a portable digital device that measures how people walk, without needing complex lab equipment. Researchers want to see if this simple device can accurately track walking changes in people with neuromuscular diseases like muscular dystrophy and spinal muscul…
Matched conditions: MYOTONIC DYSTROPHY
Phase: NA • Sponsor: Institut de Myologie, France • Aim: Knowledge-focused
Last updated Feb 23, 2026 14:53 UTC