Myotonic dystrophy type 1 (DM1) patients in a Phase 1/2 clinical trial saw gains in motor function and cognitive measures after receiving Dyne Therapeutics’ zeleciment basivarsen (z-basivarsen), previously known as DYNE-101, supporting the recent initiation of a Phase 3 study. Findings from the Phase 1/2 ACHIEVE trial…
Treatment with Duvyzat (givinostat) was associated with functional gains in boys with Duchenne muscular dystrophy (DMD) regardless of the final dose they received, according to new analyses from a Phase 3 clinical trial. The treatment also led to a slower loss of functional muscle tissue and a slower replacement…
Discussion
I’m one of those people who can get lost deep in thought. As a busy mom to many in a bustling household that could seem like chaos to others, my thoughts are often my only quiet time. I share seven children with my husband, Jason: Lexi, 24; Max, 20; Chance,…
SGT-003, an investigational gene therapy for Duchenne muscular dystrophy (DMD), worked as expected to increase levels of microdystrophin — a version of the muscle-protecting protein that’s deficient in DMD — and preserve muscle health for boys in a clinical trial, according to new data. The one-time treatment was also well…
PGN-EDODM1, Pepgen’s experimental therapy for myotonic dystrophy type 1 (DM1), was generally well tolerated in an early clinical trial, with biomarker data suggesting that the treatment is affecting its intended molecular target. Based on these early…
Becoming educated and informed about muscular dystrophy is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your muscular dystrophy journey.
There is no cure for muscular dystrophy yet, but here you can find more information about current therapeutic approaches, as well as experimental treatments that are in the pipeline.
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