Research Articles Archives

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

December 18, 2015

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Sareptaâ€™s New Drug Application (NDA) for eteplirsen, on January 22, 2016. The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the eteplirsen NDA is February 26, 2016.

BioMarin Announces That FDA Has Advised it Will Not Take Action on the Kyndrisaâ„¢ (drisapersen) New Drug Application by the PDUFA Date

December 18, 2015

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the U.S. Food and Drug Administration (FDA) has notified the Company that they had not yet completed their review process and would be unable to take an action by the Prescription Drug User Fee Act (PDUFA) action date for KyndrisaTM (drisapersen) of December 27, 2015, and anticipate taking action in early January 2016.

Catabasis Pharmaceuticals Completes Enrollment for Part A of the MoveDMDSM Trial, a Phase Â_ Trial of CAT-1004 for the Treatment of Duchenne Muscular Dystrophy

December 18, 2015

Catabasis Pharmaceuticals, Inc. (NASDAQ: CATB), a clinical – stage drug development company built on a pathway pharmacology technology platform, today announced that enrollment is complete in Part A of the MoveDMD trial, a Phase 1/2 trial of CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD).

Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders

December 10, 2015

Clustered regularly interspaced short palindromic repeat (CRISPR) has arisen as a frontrunner for efficient genome engineering. How-
ever, the potentially broad therapeutic implications are largely unexplored.

FDA Advisory Committee Discusses Clinical Data Package for BioMarin’s Kyndrisa(TM) (drisapersen) for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

November 24, 2015

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) met to discuss the data submitted to support the New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping.

BioMarin Announces Data Analysis Demonstrating Consistent Efficacy of Kyndrisaâ„¢ (drisapersen) in Comparable Patients Across Three Randomized Studies

November 20, 2015

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced data analysis for Kyndrisa demonstrating consistent evidence of efficacy in comparable patients across three randomized, placebo-controlled studies that were conducted contemporaneously. Kyndrisa is currently under review with U.S. and European health authorities for the treatment of patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.

Sarepta Therapeutics Announces Publication of Positive Long-Term Safety and Efficacy Data for Eteplirsen in the Annals of Neurology

November 17, 2015

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-based therapeutics, today announced that the Annals of Neurology published online [link to final article] positive efficacy and safety results from a Phase IIb long-term open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

Duchenne muscular dystrophy is a stem cell disease

November 16, 2015

A new study from The Ottawa Hospital and the University of Ottawa is poised to completely change our understanding of Duchenne muscular dystrophy and pave the way for far more effective treatments.

Santhera Reports New Data and Updates on Regulatory Filings for RaxoneÂ® (idebenone) in Duchenne Muscular Dystrophy (DMD)

November 11, 2015

Santhera Pharmaceuticals (SIX: SANN) announces that it has now completed comparative analyses of the respiratory outcomes for patients in its successful Phase III DELOS trial with data from a natural history DMD patient cohort collected by the Cooperative International Neuromuscular Research Group (CINRG).