The drug golodirsen, which treats Duchenne's muscular dystrophy, was accepted by the FDA and qualified for priority review

progressive muscular dMD is an X-linked genetic disease caused by mutations in the genes encoded on the X chromosome that encode antimuscular hytrophin (dystrophin)today, SareptaCompany(http://announced that its application for thenew
drughttp:// (http://(NDA) for the treatment of Duchenne's muscular dystrophy (DMD
)http:// was accepted and qualified for priority review by thefda (http://The application is expected to be approved by August 19, 2019About Golodirsen
Golodirsen is a Sarepta-proprietary phosphamide-polynucleotide, targeted by the cutting process of the antiamattrophic protein mRNA precursor, focusing on the introduction of exon 53 jumps (exon53 skipping) to produce short but functional antimyotrophin proteinaccording to , about 8% of DMD patients carry genetic mutations suitable for exogenous 53 jump therapy Previously, golodirsen was awarded fast-track status by the FDA 　　Based on the positive results shown in the 4053-101 test (http:// , Sarepta rolled out golodirsen's NDA in December 2018 The 4053-101 trial assessed the safety, tolerability, pharmacokinetic properties, and expression of antiamatphine protein in 25 DMD patients who were identified as suitable for exosome 53 jump therapy Golodirsen reached all biological endpoints, including the transcription of RNA that correctly performed the exon jump, the expression of antimuscular ostrophy protein, and the increase in muscle strength Currently, golodirsen and casimersen (introducing exon 45 jumps) are also receiving assessments in THE ESSENCE trial as a potentially validated post-marketing study