Muscular Dystrophy Insights

Parent Project Hosts 2025 Duchenne Healthcare Summit in San Diego

Parent Project Muscular Dystrophy is hosting the 2025 Duchenne Healthcare Professionals Summit in San Diego, California, to bring together experts and caregivers to discuss the latest in Duchenne muscular dystrophy care. The summit aims to improve treatment and quality of life for those with Duchenne muscular dystrophy.

Scotland Approves New Treatment for Duchenne Patients

Scotland has approved a new treatment for Duchenne muscular dystrophy, a genetic disorder that causes progressive muscle weakness. This approval brings hope for improved quality of life and potentially slower disease progression for those affected by this condition.

MDA Opens College Scholarship Applications for Students with Neuromuscular Conditions

The Muscular Dystrophy Association is now accepting applications for its college scholarship program, which supports students with neuromuscular conditions, including muscular dystrophy. The scholarships aim to help these students pursue higher education despite their health challenges.

First Female Receives Revolutionary Gene Therapy in Miami

A woman in Miami became the first female in the world to receive a revolutionary new gene therapy for muscular dystrophy, offering potential new hope for those living with the condition. This groundbreaking treatment aims to address the genetic root cause of muscular dystrophy.

Recent studies have highlighted the severe cardiac complications associated with different forms of muscular dystrophy. Blackstone et al. (2025) found that a boy with Duchenne muscular dystrophy (DMD) experienced an unusually rapid progression of cardiomyopathy due to a genetic deletion also affecting the XK gene. This suggests a more severe cardiac phenotype when DMD is combined with McLeod neuroacanthocytosis syndrome.

In the realm of treatment, Ciccone (2025) reports that Capricor has submitted a Biologics License Application for deramiocel, a potential new therapy for DMD cardiomyopathy, which may receive priority review from the FDA.

Meanwhile, research published by Sugiyama et al. (2025) on Fukuyama congenital muscular dystrophy (FCMD) indicates that while no fatal arrhythmias were observed, patients showed an increase in premature ventricular contractions with age, hinting at progressive cardiac dysfunction.