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 much milder Becker Muscular Dystrophy (BMD) phenotype.
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.
Duchenne FAQ
We have prepared an overview of the most frequently asked questions and provided answers which will hopefully clarify some of your questions about DMD.
More Information about DMD
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Parent Project Muscular Dystrophy
PPMD’s mission is to improve the treatment, quality of life, and long-term outlook for all individuals affected by Duchenne Muscular Dystrophy.
visit www.parentprojectmd.org
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Duchenne Parent Project
Dutch site for parents and patients containing information on DMD, the Duchenne Parent Project organization and its activities, and the status of recent research and care.
visit www.duchenne.nl
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MDA - Duchenne Muscular Dystrophy
MDA is dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research, providing comprehensive health care and support services.
visit www.mda.org/disease/dmd
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Leiden Muscular Dystrophy
Detailed scientific information on research and/or diagnosis in Duchenne and Duchenne-like muscular dystrophies (i.e. Duchenne, Becker, Limb-Girdle).
visit www.dmd.nl
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VSN
Dutch site aimed at providing improved standards of care, facilitating effective research, and providing advice and information to patients with a muscular disorder.
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Cure Duchenne
CureDuchenne is a non-profit organization founded in 2003. CureDuchenne aggressively seeks the most promising, leading edge research breakthroughs.
visit www.cureduchenne.org