Sarepta Therapeutics Announces FDA Will Not Complete the Review of the Eteplirsen New Drug Application By The PDUFA Date

May 25, 2016

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has notified the Company that they are continuing their review and internal discussions related to our pending NDA for eteplirsen and will not be able to complete their work by the Prescription Drug User Fee Act (PDUFA) goal date of May 26, 2016. The FDA has communicated that they will continue to work past the PDUFA goal date and strive to complete their work in as timely a manner as possible.

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DMDRP Program Announcements Released

May 24, 2016

Duchenne Muscular Dystrophy Research Program Announcements have been released

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WAVE Life Sciences to Advance Next-Generation Nucleic Acid Therapies to Address Unmet Need in Duchenne Muscular Dystrophy

May 9, 2016

WAVE Life Sciences Ltd. (NASDAQ:WVE), a genetic medicines company focused on developing stereopure nucleic acid therapies for patients impacted by rare diseases, today reaffirmed its commitment to advance next-generation nucleic acid therapies to address the significant unmet need of patients diagnosed with Duchenne Muscular Dystrophy (DMD)

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NICE Recommends Translarna™ (ataluren) for the Treatment of Patients with Nonsense Mutation Duchenne Muscular Dystrophy in England

April 15, 2016

PTC Therapeutics, Inc. (NASDAQ: PTCT), today announced that the National Institute for Health and Care Excellence (NICE) has recommended Translarna ∇ (ataluren) for ambulatory patients aged five years and older with nonsense mutation Duchenne muscular dystrophy (nmDMD) in connection with a Managed Access Agreement (MAA) with NHS England. The provision of patient access is subject to the finalization of the NICE draft guidance, which the agency expects in May of 2016.

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Capricor Therapeutics Announces DSMB Recommends Continuation of HOPE-Duchenne Clinical Trial

April 4, 2016

Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics, today announced that the independent Data Safety Monitoring Board (DSMB) of the Phase I/II HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) clinical trial has completed its pre-specified review of the first patient cohort.

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