Articles

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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CureDuchenne Extends Prosensa Pipeline Collaboration With $7 Million

August 13, 2014

Comment Now Follow Comments CureDuchenne, a California-based non-profit organization dedicated to finding a cure for Duchenne Muscular Dystrophy (DMD), announced on Tuesday their $7 million (€5 million) collaboration with the biotechnology company, Prosensa Holding N.V.

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Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice

August 10, 2014

Most mutations that truncate the reading frame of the DMD gene cause loss of dystrophin expression and lead to Duchenne muscular dystrophy

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Ataluren becomes the world’s first approved treatment for Duchenne muscular dystrophy

August 4, 2014

Today marks a much anticipated major milestone in the treatment of Duchenne, as Translarna™ (ataluren) was granted conditional marketing authorization in the European Union for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older.

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Dystrophin expression in muscles of duchenne muscular dystrophy patients after high-density injections of normal myogenic cells.

July 24, 2014

The transplantation of myoblasts obtained from a healthy donor is a potential treatment of Duchenne muscular dystrophy (DMD). Following intramuscular injection, donor myoblasts can fuse with the myofibers of the patient and introduce the normal dystrophin gene. In a previous Phase 1A clinical trial, (Dystrophin expression in muscles of DMD patients after high-density injections of normal myogenic cells, http://www.ncbi.nlm.nih.gov/pubmed/16691118?dopt=Abstract) the investigators showed that transplantation of myoblasts grown from the muscle biopsy of a healthy donor introduced the normal dystrophin gene in the DMD myofibers, with the consequent expression of the normal dystrophin mRNA and restoration of the dystrophin protein in several myofibers.

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