Sarepta Therapeutics’ SRP-9001 (delandistrogene moxeparvovec) has received the backing of the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) of the US Food and Drug Administration (FDA) for accelerated approval for Duchenne muscular dystrophy (DMD). The FDA committee voted 8 to 6 in favor of the investigational gene therapy for the treatment of ambulatory patients […]
US-based PTC Therapeutics has secured marketing approval in Russia for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The approval for Translarna has been given by the Ministry of Health of the Russian Federation for Duchenne muscular dystrophy caused by a nonsense mutation in patients, aged two years and older. Stuart […]
Astellas acquisition of Audentes: Japanese pharma company Astellas Pharma has agreed to acquire US gene therapy company Audentes Therapeutics in an all-cash deal worth about $3 billion, as per pharma acquisition news. Based in California, Audentes Therapeutics calls itself a clinical-stage company focused on developing a portfolio of Adeno-associated virus (AAV)-based genetic medicines for the […]
DailyHealthNeeds.com brings you the basic introduction to Muscular Dystrophy in simple easy to understand language. Find out about Muscular Dystrophy causes, Muscular Dystrophy symptoms and Muscular Dystrophy types to get awareness of this fatal disease.
DailyHealthNeeds.com brings you the basic introduction to Muscular Dystrophy in simple easy to understand language. Find out about Muscular Dystrophy causes, Muscular Dystrophy symptoms and Muscular Dystrophy types to get awareness of this fatal disease. Muscular Dystrophy is a rare muscular disease that usually occurs in young boys. This genetic disorder which is also referred […]
US based Exonics Therapeutics has secured $5 million seed funding from CureDuchenne Ventures, a subsidiary of nonprofit DMD advocacy group, CureDuchenne to advance its therapy for Duchenne Muscular Dystrophy, a severe form of muscular dystrophy.
Exonics Therapeutics, a biotechnology firm based in Boston, Massachusetts, has secured $5 million in seed funding from CureDuchenne Ventures, a subsidiary of the nonprofit Duchenne Muscular Dystrophy (DMD) advocacy group, CureDuchenne. This funding is aimed at further developing gene editing technologies, including CRISPR/Cas9, to potentially offer a curative therapy for Duchenne Muscular Dystrophy, a severe […]
Pathological conditions related to the muscular system are: Muscular dystrophy Polymyositis Muscular dystrophy: Muscular dystrophy is a group of inherited diseases characterized by progressive weakness and degeneration of muscle fibers without the involvement of the nervous system. Polymyositis: Polymyositis is a chronic inflammatory myopathy. This condition is marked by symmetrical muscle weakness and pain, often […]