Congenital myotonic dystrophy
MONDO:0023595Myotonic dystrophy that is present at birth.
Also known as: congenital myotonic dystrophy, Congenital Myotonic dystrophies, Congenital Myotonic dystrophy, Congenital myotonic dystrophy, MYOTONIC dystrophy CONGEN, Myotonic dystrophies, Congenital, Myotonic dystrophy, Congenital, dystrophies, Congenital Myotonic
14 clinical trials for this condition and its sub-types, 9 tagged with Congenital myotonic dystrophy itself.
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New drug shows promise for rare muscle disease in kids
Disease control CompletedThis study tested a drug called tideglusib against a placebo in 56 children aged 6 to 16 with congenital myotonic dystrophy, a rare genetic muscle disorder. The goal was to see if tideglusib could reduce symptoms like muscle weakness and breathing problems. The trial was complete…
Phase 2/3 • Sponsor: AMO Pharma Limited • Aim: Disease control
Last updated Jun 27, 2026 07:54 UTC
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Experimental drug tideglusib tested for rare muscle disease
Disease control CompletedThis phase 2 study tested the safety and effectiveness of tideglusib, an experimental drug, in 16 adolescents and adults with congenital or juvenile-onset myotonic dystrophy type 1. Participants received either 400 mg or 1000 mg of tideglusib daily. The study measured side effect…
Phase 2 • Sponsor: AMO Pharma Limited • Aim: Disease control
Last updated Jun 26, 2026 16:48 UTC