Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious, and other diseases.
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|Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping|
--Advisory Committee Meeting Rescheduled for
--PDUFA date for eteplirsen is
It is estimated that Duchenne muscular dystrophy affects approximately one in every 3,500 – 5,000 boys born worldwide, with 13% of people with the disease having mutations addressable by eteplirsen/exon 51 skipping.
About Sarepta Therapeutics
Eteplirsen is designed to address the underlying cause of DMD by
restoring the dystrophin messenger RNA (mRNA) reading frame, thus
enabling the production of a shorter, functional form of the dystrophin
protein. Eteplirsen uses Sarepta’s proprietary phosphorodiamidate
morpholino oligomer (PMO) chemistry and exon-skipping technology to skip
exon 51 of the dystrophin gene. Approximately 13 percent of the DMD
population is amenable to exon 51 skipping. Data from clinical studies
of eteplirsen in DMD patients have demonstrated a consistent safety and
tolerability profile and have also shown measurable dystrophin protein
expression. Promoting the synthesis of a shorter dystrophin protein is
intended to slow the decline of ambulation and mobility seen in DMD
patients. There currently is no approved treatment in
About Duchenne Muscular Dystrophy
DMD is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500-5,000 boys worldwide. A devastating and incurable muscle-wasting disease, DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Progressive muscle weakness in the lower limbs spreads to the arms, neck and other areas. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and death usually occurs before the age of 30.
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