December 12, 2019: The U.S. Food and Drug Administration granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) for the patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping.
It is estimated that approximately 8 per cent of patients with DMD have this mutation. This is the new medical treatments for serious neurological disorders for the treatments for rare diseases.
DMD is a rare genetic disorder that is characterized by progressive muscle deterioration and weakness. It is caused by the absence of dystrophin, a protein that helps keep muscle cells intact.
Patients  with DMD progressively lose the ability to perform activities separately and often require a wheelchair by their early teens.
Vyondys 53 approved under the accelerated approval pathway that provides for the approval of drugs, treat serious or life-threatening diseases and generally offer a meaningful advantage over existing treatments. https://fda.einnews.com/pr_news/504734147/fda-grants-accelerated-approval-to-first-targeted-treatment-for-rare-duchenne-muscular-dystrophy-mutation
