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 ...
Advanced imaging and theoretical physics unlock clues to new treatments for muscular dystrophy
A new discovery about how tiny protein clusters form in cells could pave the way for treatments for Emery-Dreifuss muscular ...
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.
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 ...
Science Daily6d
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 ...
FDA Accepts Biologics License Application for Duchenne Muscular Dystrophy Cardiomyopathy Treatment
NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that acceptance has been received by Capricor Therapeutics, Inc. (Headquarters: California, ...
Business Wire2d
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 ...
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 ...
The Cardiology Advisor3d
Deramiocel Granted Priority Review for DMD Cardiomyopathy
The FDA has granted Priority Review to the BLA for deramiocel (CAP-1002) for the treatment of Duchenne muscular dystrophy cardiomyopathy.
Business Wire7d
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 ...
Morningstar2d
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 ...