Meet the La Jolla researcher who helped discover new muscular dystrophy drug
Having found that in biomedical research, the Bird Rock resident and scientist at La Jolla’s Sanford Burnham Prebys has ...
Onlymyhealth5h
FDA Approves First-Ever Myasthenia Gravis Treatment For Children—A Game Changer In Pediatric Care
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!
Science Daily7d
Scientists unlock clues to new treatments for muscular dystrophy
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 ...
Solid Biosciences to Present at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from the Phase 1/2 INSPIRE DUCHENNE ...
Dyne: Accelerated Approval Of DYNE-101 Might Be Possible With Surrogate Biomarker
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.
Business Wire4d
Edgewise Therapeutics to Present on Sevasemten for the Treatment of Becker Muscular Dystrophy at the 2025 MDA Clinical and Scientific Conference
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 ...
Independent.ie on MSN1d
Kerry staff come together to help seven-year old Archie get treatment
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 ...
FDA Approves Expanded Use of Eculizumab (Soliris) for Pediatric Myasthenia Gravis, Bringing New Treatment Options for Children
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 ...
Business Wire8d
Italfarmaco Provides Overview on Givinostat as Treatment for Duchenne Muscular Dystrophy: Progress in Global Access, Regulatory Milestones and Clinical Trials
Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, the company’s drug for the treat ...
Morningstar4d
Edgewise Therapeutics to Present on Sevasemten for the Treatment of Becker Muscular Dystrophy at the 2025 MDA Clinical and Scientific Conference
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 to Present on Sevasemten for the Treatment of Becker Muscular Dystrophy at the 2025 MDA Clinical and Scientific Conference
Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on sevasemten, an investigational orally ...