Duchenne muscular dystrophy is an inherited disorder that involves muscle weakness that worsen quickly and is a fatal genetic disorder diagnosed in the childhood. The fatal disease is caused by the mutation in the Duchenne gene found on the X-chromosome that encodes for dystrophin. The disease progression leads to muscle weakness and serious medical problems including issues related to the heart and lungs. It has been observed that young men with Duchenne muscular dystrophy live only till late twenties. The symptoms associated with Duchenne muscular dystrophy include fatigue, fever with cough, learning difficulties, intellectual disability, muscle weakness, difficulty with motor, frequent falls, trouble getting up from a lying position or climbing stairs, progressive difficulty in walking. There is no cure for Duchenne muscular dystrophy and treatment mainly aims to control symptoms to improve quality of life.
The drug candidates in the pipeline of Duchenne muscular dystrophy include, but are not limited to, DS-5141, BMS-986089 and SRP-4053. Some of the companies having drugs in the Duchenne muscular dystrophy pipeline include GTx, Inc., Sarepta Therapeutics Inc., Santhera Pharmaceuticals Holding, and Marina Biotech, Inc.
The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with detailed analysis of pipeline and clinical trials. Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licencing, grants, technology and others.