"In many countries, health insurance changes when DMD men are no longer seen in a specialized pediatric center. This impacts massively on the quality of care they receive. For this reason, many DMD ...

Columnist Shalom Lim is using his lived experience with Duchenne muscular dystrophy to consult with organizations about ...

Gene therapies have extended survival in muscular dystrophy, requiring improved care transitions from pediatric to adult systems. Current systems lack adequate staffing, specialists, and communication ...

Investigators based in Italy recently assessed the impacts of swallowing difficulties in a cohort of patients with DMD who presented to a single hospital with respiratory issues. The Swallowing ...

Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription. Patient advocacy organizations often bring ...

Data are scarce regarding the benefits of strength training for muscular dystrophies, for which there are no cures. Strength training to ameliorate the effects of muscular dystrophies (MDs) was found ...

A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 -- the most prevalent form of adult-onset muscular dystrophy -- provides insight into why patients develop fatty liver ...

On Monday, the U.S Food and Drug Administration (FDA) lifted the clinical hold on Entrada Therapeutics, Inc. (NASDAQ:TRDA) ENTR-601-44. The agency issued the hold in December 2022. In November 2023, ...

PGN-EDODM1, an experimental DM1 therapy from Pepgen, showed biomarker activity and splicing correction in an early clinical ...

The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...

– Significant decreases were observed in circulating levels of creatine kinase (CK) and fast skeletal muscle troponin I (TNNI2), biomarkers associated with skeletal muscle damage – – Sevasemten was ...