Articles

Sarepta Therapeutics Announces Regulatory Update on Eteplirsen

October 27, 2014

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-based therapeutics, today provided an update on its discussions with the U.S. Food and Drug Administration (FDA) regarding its planned New Drug Application (NDA) submission for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD).

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Confirmatory Study of Eteplirsen in DMD Patients

September 29, 2014

The main objective of this study is to provide confirmatory evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety and biomarkers.

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Prosensa publishes a sensitive, reproducible and objective methodology for dystrophin analysis in patients with Duchenne muscular dystrophy

September 24, 2014

Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that the results of its research into developing an accurate and reproducible method for the measurement of dystrophin in patients with Duchenne muscular dystrophy (DMD) and Becker's muscular dystrophy (BMD) have been published in the online peer reviewed journal PLOS ONE (http://bit.ly/ZJ8ShM).

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Prosensa announces commencement of re-dosing of drisapersen in North America in patients with Duchenne muscular dystrophy

September 17, 2014

Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that a comprehensive program of re-dosing has commenced, with the first patients now re-dosed in the United States. All dosing in the drisapersen clinical program had been placed on hold by GSK on September 20, 2013, upon announcement of the DEMAND III study results.

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