Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease that affects 1 in every 3,500 to 5,000 male neonates worldwide. It is caused by mutations in the gene encoding the protein dystrophin. These genetic changes emerge as developmental delays and, in more severe types of DMD, limb weakness, loss of indep...
Find MoreSarepta Therapeutics, a leading player focused in developing precision genetic medicines for rare diseases with more than 25 programs ongoing, has recently announced the submission of New Drug Application (NDA) to the US FDA for Casimersen (SRP-4045) for the treatment of Duchenne muscular dystrophy (DMD) with a gen...
Find MoreThe Duchenne Muscular Dystrophy Market Size (DMD Market) in the 7MM was found to be USD 266.06 Million in 2017, during the study period (2017-2030). A rare muscle disorder, Duchenne Muscular Dystrophy (DMD), approximately affects 1 in 3,500 male births globally. Moreover, Duchenne Muscular Dystrophy prevalence ...
Find MoreBefore approving an investigational drug, safety and effectivity needs to be proven. Something different happened with Sarepta’s Muscular dystrophy drug Eteplirsen, as its luck changed completely when the U.S. Food and Drug Administration on 19th September 2016 approved Sarepta Therapeutics' Exondys 51 (eteplirsen) ...
Find MoreGene Therapy to “fix” Genetic disorders! "We used to think that our fate was in our stars, but now we know that, in large measure, our fate is in our genes, "quotes James Watson. This fate and our future is locked in our genes, and Gene therapy is unlocking these doors. Many physicians are predicting that in twent...
Find MoreThe American Society of Clinical Oncology (ASCO) is one of the largest and most respected conferences in the field of oncology. Held annually, this conference brings together researchers, physicians, and other healthcare professionals from around the world to discuss the latest advances in cancer research, diagnosis, and treatment.