… 47;2a Clinical Treatment Study ongoing in Oculopharyngeal Muscular Dystrophy Patients (OPMD) … future discussions with the FDA as we work toward … -301 has received Orphan Drug Designation from the EMA … Orphan Drug and Fast Track Designations from the FDA. About …

… recently hosted its 15th Annual Muscular Dystrophy Association (MDA) bowling event, raising … treatments have been approved by the U.S. Food and Drug Administration (FDA … ) to treat various neuromuscular diseases, with …

… for Capricor Therapeutics’ Duchenne muscular dystrophy (DMD) cardiomyopathy gene … development officer at Deciphera Pharmaceuticals. “That lasted … of the FDA’s Center for Drug Evaluation and … Subscribe to ClinicaSpace! Clinical trial results, research news, …

… , using the same trial process, regulatory authorities … is showing promise for treating pancreatic cancer, lung … by the innovative pharmaceutical industry. They also … treatments for Alzheimer’s and muscular dystrophy. The FDA is responsible for ensuring drug …

… 2 MARINA® Trial  The MARINA® trial was a … are no approved drugs for people living … DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). … treatment option for DM1 and become the first globally approved drug to treat …

Abstract  Human gait represents an integrated motor function governed by cortical, subcortical,  cerebellar, brainstem, spinal, and peripheral sensory systems. Abnormal gait patterns  frequently serve as early indicators of neurological dysfunction …

In India, rare diseases are not so rare. It is estimated that around 70 million Indians live with one of more than 7,000 rare diseases, many of which are genetic in origin.  A 22-year review from a major Indian genetics center found that of more than 7,000 …

… by the FDA in 2023, Regenxbio is developing its treatment. Regenxbio filed … ) regarding the latter’s Duchenne muscular dystrophy (DMD) treatment. According to a Reuters … . Food and Drug Administration in 2023, Regenxbio is developing its treatment. Regenxbio …

… chemist honoured for helping create drug to treat ALS Dane developed a … their diagnosis, according to the Muscular Dystrophy Association. However, some patients can …

… Consulting. Teijin Pharma and Aska Pharmaceutical entered into a … the FDA declined to approve its drug to treat a … application for its Duchenne muscular dystrophy treatment. Compass Pathways said … its Phase 3 dwarfism drug trial succeeded. Citius Oncology …