MYOTONIC DYSTROPHY TYPE 1 (DM1)
Clinical trials for MYOTONIC DYSTROPHY TYPE 1 (DM1) explained in plain language.
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New hope for DM1: VX-670 safety trial expands
Disease control ENROLLING_BY_INVITATIONThis study looks at the long-term safety and effects of the drug VX-670 in 44 adults with myotonic dystrophy type 1 (DM1). All participants must have already taken VX-670 in an earlier study. Researchers will monitor side effects and measure drug levels in the blood. This is an o…
Matched conditions: MYOTONIC DYSTROPHY TYPE 1 (DM1)
Phase: PHASE2 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 26, 2026 07:16 UTC
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New drug aims to ease muscle stiffness in rare disease
Disease control OngoingThis phase 3 study tests an experimental drug called AOC 1001 for myotonic dystrophy type 1, a genetic condition that causes muscle weakness and stiffness. About 159 adults who can walk independently are receiving either the drug or a placebo to see if it improves hand function, …
Matched conditions: MYOTONIC DYSTROPHY TYPE 1 (DM1)
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 26, 2026 07:06 UTC