DMD - Duchenne Muscular Dystrophy
The severe and progressive deterioration of muscle fibers in Duchenne Muscular Dystrophy (DMD) is caused by mutations in the DMD gene, mostly deletions of one or more exons, that disrupt the open reading frame of the transcript and prematurely abort the synthesis of the dystrophin protein. Using Prosensa’s RNA-modulating therapeutics, specific exon skipping can be induced during pre-mRNA splicing by disturbing specific exon inclusion signals. Mutation-specifically designed skipping of one or more exon(s) in DMD patients allows restoration of the mutated open reading frame, introduction of novel dystrophin synthesis, and conversion of a severe DMD into a typically milder Becker Muscular Dystrophy (BMD) phenotype.
The Frequently Asked Questions section contains some examples of how the exon skipping technique aims to work, as well as many other questions that Prosensa often receives.
Overview clinical symptons DMD
DMD Patient Organizations
Prosensa has established strong partnerships with organizations which take care of the interests of DMD patients and their families.
We have prepared an overview of the most frequently asked questions and provided answers which will hopefully clarify some of your questions about DMD.
Exon skipping is a highly specific technique. Therefore, it cannot be applied to all sub-populations of DMD patients. To know if exon skipping could potentially help, it is essential to…
The lead product, drisapersen (PRO051/GSK2402968), is currently involved in several clinical studies. PRO044, which aims at a different sub-population of DMD patients, is in a Phase I/IIa clinical study and PRO045 and PRO053 are anticipated to enter the clinic in the first half of 2013.