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Sarepta Therapeutics Announces Regulatory Update on Eteplirsen
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).
Confirmatory Study of Eteplirsen in DMD Patients
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.
Prosensa publishes a sensitive, reproducible and objective methodology for dystrophin analysis in patients with Duchenne muscular dystrophy
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).
Prosensa announces commencement of re-dosing of drisapersen in North America in patients with Duchenne muscular dystrophy
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.
The intracellular Ca2+ channel MCOLN1 is required for sarcolemma repair to prevent muscular dystrophy.
The integrity of the plasma membrane is maintained through an active repair process, especially in skeletal and cardiac muscle cells, in which contraction-induced mechanical damage frequently occurs in vivo.
PTC Therapeutics, Inc. completes enrollment of landmark trial in Duchenne muscular dystrophy
PTC Therapeutics, Inc. today announced that it has completed enrollment of ACT DMD, the Phase 3 confirmatory trial of Translarna (ataluren) for patients with nonsense mutation Duchenne muscular dystrophy (nmDMD).
Prosensa announces Lancet Neurology publication of an exploratory phase II study (DEMAND II) demonstrating efficacy and safety of drisapersen in patients with Duchenne muscular dystrophy
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 full data from its exploratory, double-blind, placebo-controlled Phase II study (DEMAND II/ DMD114117/ NCT01153932) of drisapersen in patients with Duchenne muscular dystrophy (DMD) have been published in Lancet Neurology http://bit.ly/1rSMpeJ.
Prosensa is working towards long-term access to drisapersen
As previously communicated, Prosensa received guidance from the United States Food and Drug Administration (FDA) on June 2, in which a regulatory pathway for accelerated approval was outlined for drisapersen.