Articles

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 The provision of patient access is subject to the finalization of the NICE draft guidance, which the agency expects in May of 2016.

Read more

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.

Read more

Catabasis Pharmaceuticals Presents Positive CAT-1004 Data from Part A of the MoveDMDSM Trial at the 2016 Muscular Dystrophy Association Clinical Conference

March 21, 2016

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced positive data from Part A of the MoveDMD trial of CAT-1004 for Duchenne muscular dystrophy (DMD).

Read more

PTC Therapeutics Provides Update on Health Canada Review of Translarna™ (ataluren) for the Treatment of Duchenne Muscular Dystrophy

March 14, 2016

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company intends to submit the results of the recently completed Phase 3 ACT DMD study for review by Health Canada as part of the New Drug Submission (NDS) for Translarna™ (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD). In order to submit this additional data for review, PTC will withdraw the current NDS from Health Canada and resubmit the NDS with the ACT DMD results. As a result, the company no longer expects Health Canada review of the NDS in the first half of 2016.

Read more

Sarepta Therapeutics Announces FDA Advisory Committee Meeting to Review Eteplirsen as a Treatment for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

March 14, 2016

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Sarepta’s New Drug Application (NDA) for eteplirsen on April 25, 2016. The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the eteplirsen is May 26, 2016.

Read more
Items 1 - 5 of 128  12345678910Next