Articles

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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The intracellular Ca2+ channel MCOLN1 is required for sarcolemma repair to prevent muscular dystrophy.

September 14, 2014

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.

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PTC Therapeutics, Inc. completes enrollment of landmark trial in Duchenne muscular dystrophy

September 9, 2014

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).

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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

September 8, 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 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.

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Prosensa is working towards long-term access to drisapersen

August 15, 2014

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.

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