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19.09.2016-Sarepta Therapeutics Announces FDA Accelerated Approval of EXONDYS 51™ (eteplirsen) injection, an Exon Skipping Therapy to Treat Duchenne Muscular Dystrophy (DMD) Patients Amenable to Skipping Exon 51.


  • The drug will be useful for DMD children with a confirmed deletion/mutation of Exon 51 only.
  • Commercial sale of the drug will be launched soon in the U.S.

About EXONDYS 51™ (Formerly known as, ‘Eteplirsen’)

EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. Data from clinical studies of EXONDYS 51 in a small number of DMD patients have demonstrated a consistent safety and tolerability profile. The pivotal trials were not designed to evaluate long-term safety and a clinical benefit of EXONDYS 51 has not been established.

Please visit http://www.exondys51.com/ for more information about the drug.


FDA has accepted the NDA for Eteplirsen

Eteplirsen, the lead clinical candidate of Sarepta Therapeutics, is designed to skip exon 51. A candidate for exon 53 skipping (SRP-4053) is in clinical development, and a candidate for exon 45 skipping (SRP-4045) has entered early clinical development. Sarepta also have other drug candidates in discovery and preclinical development that are designed to skip exons 44, 52, 50, 43, 55, 8 and 35, and they are committed to exploring the potential of their technology to address all DMD patients who may be candidates for exon skipping.

Sarepta Therapeutics Announces Tentative FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Oct. 14, 2015-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) is tentatively scheduled to review Sarepta’s New Drug Application (NDA) for eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, on January 22, 2016. There has been no formal communication to the company or Federal Register notice of a confirmed advisory committee meeting date.

It is estimated that DMD affects approximately one in every 3,500 boys born worldwide, with 13% of people with the disease having mutations addressable by eteplirsen/exon 51 skipping.

“We have been verbally informed by the FDA that we have a tentative date of January 22, 2016 for our advisory committee review of eteplirsen and our PDUFA date of February 26, 2016, remains unchanged.” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “We look forward to discussing the efficacy and safety data included in our NDA submission for eteplirsen with the committee, with the ultimate goal of bringing treatment to more patients with Duchenne.”

About Sarepta Therapeutics

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. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates, including its lead DMD product candidate, eteplirsen, designed to skip exon 51. Sarepta is also developing therapeutics for the treatment of infectious diseases, such as drug-resistant bacteria and other rare human diseases. For more information, please visit us at www.sarepta.com.

About Eteplirsen

Eteplirsen is Sarepta's lead drug candidate and is designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression.

Eteplirsen uses Sarepta's novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect approximately 13% of the total DMD population. By skipping exon 51, eteplirsen may restore the gene's ability to make a shorter, but still functional, form of dystrophin from messenger RNA, or mRNA. Promoting the synthesis of a truncated dystrophin protein is intended to stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD.