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Sarepta Therapeutics, Inc. Message Board

  • psmith0150 psmith0150 Dec 20, 2012 10:17 PM Flag

    The FDA has 60 days to respond to a fast track request. When did SRPT file their request???

    Does any one know the date??

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    • Eteplirsen: A Novel Gene Therapy for the Treatment of DMD

      For the first time, promising new disease-modifying therapies such as eteplirsen are being developed that can bring potentially life-saving treatments to subpopulations of DMD patients with different deletions in the dystrophin gene. These therapies have the potential to fulfill the significant unmet need for these patients.

      A particularly promising approach to the treatment of DMD involves the use of a technique known as exon-skipping, which was developed by scientists as a way to potentially restore the functionality of the dystrophin gene. Eteplirsen uses AVI BioPharma’s proprietary exon-skipping technology. This involves realigning the genetic information in the defective dystrophin gene by “skipping over” the missing piece of DNA (i.e., exon 51) so that protein synthesis can proceed. It results in the production of a shortened, yet functional, version of dystrophin. Researchers at the company believe that promoting synthesis of a shortened dystrophin protein might improve, stabilize, or significantly slow the progression of DMD. It may also prolong and improve the quality of life for specific patients with the disease. In addition to eteplirsen, AVI BioPharma is currently evaluating other exon-skipping drug candidates for the treatment of DMD.

      The FDA and the EMA granted etelplirsen orphan drug status in November 2007 and December 2008, respectively. The FDA also granted eteplirsen fast track status in December 2007.

    • This Sarepta drug already has fast track ,do you mean early approval?

    • only the shadow knows

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