Articles

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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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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