Dyne Reports One-Year DM1 Trial Improvements
Dyne Therapeutics reported one-year Phase 1/2 ACHIEVE trial data showing sustained improvements in measures of myotonia, muscle strength, mobility and patient-reported health among people with myotonic dystrophy type 1 taking investigational z-basivarsen. The company said the treatment had a favorable safety profile; registrational expansion results are expected in 2027 and could support a U.S. accelerated approval application.
Health · 8 sources · 5dRead itThe journal
Open the journal →The conversation starts in the journal — be the first to post.
About myotonic dystrophy
Myotonic dystrophy (DM) is a type of muscular dystrophy, a group of genetic disorders that cause progressive muscle loss and weakness. In DM, muscles are often unable to relax after contraction. Other manifestations may include cataracts, intellectual disability and heart conduction problems. In men, there may be early balding and infertility. While myotonic dystrophy can occur at any age, onset is typically in the 20s and 30s.
Everything about myotonic dystrophy →