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Drisapersen Clinical Trial Program Duchenne Muscular Dystrophy Changes in the dystrophin gene (mutations) that lead to the near absence of dystrophin protein result in the most severe form of The largest and longest Duchenne clinical trial program submitted to the FDA. dystrophin deficient muscular dystrophy, Duchenne muscular dystrophy, also known as just Duchenne. Since 2007 It is estimated to be present in 1 in 3,500-5,000 newborn boys with symptoms appearing as early as the age of two. Dystrophin protein plays an important structural role in the performance of muscles. Without dystrophin, boys living with Duchenne experience progressive muscle weakness, causing serious medical complications including serious heart or 326 boys have participated in clinical trials with drisapersen 9clinical studies, 70sites 3randomized 25countries In More than in including placebo-controlled studies respiratory-related complications, resulting in death in early adulthood. Drisapersen & Exon 51 Skipping Drisapersen is an antisense oligonucleotide that induces exon skipping to provide a molecular patch for dystrophin transcripts produced by certain mutated dystrophin genes. Exons are the parts of a gene that contain the instructions for generating a protein. In applicable cases, skipping an exon near the mutation allows for the production of a truncated but functional dystrophin protein. Representing nearly 500 total patient years on drisapersen Drisapersen was granted Breakthrough Therapy designation by the United States Food and Drug Administration (FDA) in June 2013 And granted Orphan status in the U.S., E.U., Australia and Japan