Duchenne muscular dystrophy - Wikipedia, the free encyclopedia Duchenne muscular dystrophy (DMD) is a recessive X-linked form of muscular dystrophy, affecting around 1 in 3,600 boys, which results in muscle degeneration and eventual death.[1] The disorder is caused by a mutation in the dystrophin gene, the largest ge
Duchenne Muscular Dystrophy | MDA MDA leads the search for treatments and therapies for Duchenne muscular dystrophy (DMD). The Association also provides comprehensive supports and expert clinical care for those living with DMD. In this section, you’ll find up-to-date information about Duc
Overview | Duchenne Muscular Dystrophy | MDA What is Duchenne muscular dystrophy? Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It is one of nine types of muscular dystrophy. DMD is caused by an absence of dystrophin, a ...
Cure Duchenne - CureDuchenne: Home Exon skipping drugs will be used to transform Duchenne muscular dystrophy into a much milder disease as ...
Duchenne muscular dystrophy - FierceBiotech After a late-stage failure and a kiss-off from partner GlaxoSmithKline, Prosensa and its innovative treatment for Duchenne muscular dystrophy seemed to be on a downward slope. But now, after a series of "positive" meetings with the FDA, the company says i
News - Parent Project Muscular Dystrophy - EndDuchenne.org - Leading the fight to end Duchenne muscu What's Happening in the Duchenne Community Archive ... Trust it or Trash it? PPMD uses tools like Trust it or Trash it? to be able to produce the most accurate information possible. This is a tool to help you think critically about the quality of health i
Treatment of Duchenne and Becker muscular dystrophy Emery AE. The muscular dystrophies. Lancet 2002; 359:687. Angelini C. The role of corticosteroids in muscular dystrophy: a critical appraisal. Muscle Nerve 2007; 36:424. Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscula
Targeting mRNA Splicing as a Potential Treatment for Duchenne Muscular Dystrophy - Ryszard Kole - Di Several clinical trials have recently demonstrated that oligonucleotide-based drugs induced targeted exon skipping in dystrophin pre-mRNA in Duchenne muscular dystrophy patients, resulting in novel expression of a truncated but functional isoform of the d
New hope for Duchenne muscular dystrophy drug | Fox News The agency has now indicated that safety and efficacy data from studies without placebo groups could support an approval application, analysts said. The regulator had earlier said that the company would need to compare the drug, eteplirsen, with a placebo
Sarepta to Seek FDA Approval For Duchenne Muscular Dystrophy Drug | Xconomy Today, the Cambridge, MA-based biotech indicated that it is going to go for it. Sarepta is announcing that it plans to submit a new drug application to the FDA for its Duchenne treatment, eteplirsen, during the first half of 2014. The FDA has said that is
