The parents of an eight-year-old boy with Duchenne muscular dystrophy are pleading with doctors to give their son a drug that ...

Having found that in biomedical research, the Bird Rock resident and scientist at La Jolla’s Sanford Burnham Prebys has ...

Researchers combined advanced imaging techniques and theoretical physics to observe and explain how nanoclusters of the protein emerin form inside living cells. The study uncovers the molecular 'rules ...

The FDA has approved Soliris the first-ever treatment for children with generalized myasthenia gravis gMG offering new hope in pediatric neuromuscular care Learn more!

Dyne Therapeutics’ DYNE-101 shows positive results in phase 1/2 ACHIEVE study. Read why DYN stock may rise with FDA approval and a $2.78B market opportunity.

Prediction of North Star Ambulatory Assessment trajectories in Becker muscular dystrophy: Model development and validation Title: P11: Two-year sevasemten treatment outcomes in Becker muscular ...

The Muscular Dystrophy Association (MDA) welcomes the U.S. Food and Drug Administration (FDA) approval of the expanded indication of Alexion/AstraZeneca’s eculizumab (Soliris) for pediatric patients ...

Seven-year-old Archie Ennis has touched the hearts of the nation as his family plea for funds to help him get the life-saving ...

Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, the company’s drug for the treat ...

Edgewise Therapeutics to Present on Sevasemten for the Treatment of Becker Muscular Dystrophy at the 2025 MDA Clinical and Scientific Conference – Company to host an Industry Forum to discuss ...

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on sevasemten, an investigational orally ...