Viltolarsen targets dystrophin gene mutations, enabling functional dystrophin protein production, crucial for muscle health in patients with DMD. Consistent viltolarsen treatment slowed disease ...

Reduced progression of myocardial fibrosis measured by LGE on cardiac MRI (p=0.022)Significant improvement in LVEF versus placebo in patients ...

"While we did see correlations between activity levels and cardiomyopathy progression in patients with Duchenne muscular dystrophy, these correlations were not particularly strong. Our interpretation ...

Cardiomyopathy is the leading cause of mortality in patients with DMD. Compared with their healthy counterparts, patients with DMD — including those with preserved left ventricular ejection fraction ...

Capricor Therapeutics has presented positive long-term data from its HOPE-2 clinical trial at the 2025 Muscular Dystrophy Association Conference, indicating that its leading treatment, deramiocel, can ...

Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...

NS Pharma, Inc. (NS Pharma, New Jersey, USA; President, Yukiteru Sugiyama ), a biopharmaceutical leader in rare diseases and subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today ...

QMUS may serve as a reliable, non-invasive biomarker for monitoring DMD progression and functional status, addressing limitations of traditional assessments. Strong correlations were found between ...

Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...

"Fast Track designation is an important regulatory milestone for PBGENE-DMD and reflects the significant unmet need in DMD," said Michael Amoroso, Chief Executive Officer of Precision BioSciences. "We ...

Early screening for Duchenne muscular dystrophy can help you get early treatments and referrals for your child to help prevent further loss of muscle tissue. Duchenne muscular dystrophy (DMD) is a ...