DMD Innovative Therapy, which is eligible for FDA Priority Review, is expected to be approved in February next year.

DMD is a rare and fatal neuromuscular genetic disease that causes about one in every 3,500 to 5,000 men worldwide.
DMD is caused by a change or mutation in the gene that encodes antimyostrophy proteins.
usually occur in inf and young children.
children may experience stunting, such as difficulty walking, climbing stairs, or standing from their seats.
with the development of the disease, the lower limb muscles are unable to spread to the arms, neck and other parts.
most patients need to use a wheelchair as teenagers and then gradually lose the ability to perform daily activities independently.
, heart dysfunction can lead to heart failure due to increased breathing difficulties caused by respiratory dysfunction.
the disease is widespread and fatal, and patients usually die in their 20s.
Casimersen uses Sarepta's proprietary phosphate diamide oligonucleotide (PMO) chemical technology to design oligonucleotides.
by binding to the RNA pregenitors of the DMD gene, it alters the processing of mRNA, causing the exon 45 to jump, resulting in a truncated but still functional antimyostrophy protein.
can treat most DMD patients with exoscopic jumps (Photo: Sarepta.com) This NDA includes data from the ESSENCE study Casimersen group, a global, randomized double-blind, placebo-controlled Phase 3 clinical trial that evaluated the efficacy and safety of casimersen in patients suitable for exon 45 jump therapy.
analysis by ESSENCE showed a statistically significant increase in the production of antimyostrophy protein in the muscles of patients treated with casimersen compared to baselines and placebos.
this study is ongoing and remain blind to collect additional efficacy and safety data.
: s1. Sarepta Therapeutics Announces FDA Acceptance of Casimersen (SRP-4045) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45. Retrieved August 25, 2020, from.