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What we hope is that at the $25 million mark, the next acquirer will believe it could better serve the DMD customers under its umbrella than as a stand-alone," says Spencer.
5 letter to NATURE, suggest people with DMD who show mental retardation may have trouble producing the brain type of dystrophin, comments Donald S.
According to Niche Markets and Rare Diseases: Muscular Dystrophy, Decision Resources forecasts the launch of four new agents to treat DMD by 2021.
The planned Phase 2 trial will be a double-blind, randomized, placebo-controlled safety and efficacy study of TXA127 in 45 ambulant patients with DMD, conducted for 48 weeks, followed by a 96-week open-label extension study.
I've worked with patients with DMD for many years and this is the first time we can say with confidence that we've made a significant breakthrough towards finding a targeted treatment.
However, says Hoffman, the membrane defects themselves are probably not the cause of muscle wasting in individuals with DMD.
Today, DMD announced that it has reorganized its organization to focus primarily on three industry sectors:
Marketing professionals who have been frustrated by the lack of trustworthiness and timeliness of their HCP data now have a way to bring together data previously cocooned in disparate applications and combine it with high quality data provided by DMD," said Reltio CEO Manish Sood.
Looking beyond DMD, we hope that we can eventually pinpoint other genetic markers in individuals susceptible to developing heart failure and treat them before the first signs of heart disease in order to delay or even prevent the onset of heart failure," concludes Professor Duboc.
GSK2402968, an antisense oligonucleotide which induces exon skipping of exon 51, is currently in late stage development for DMD.
In this study, injections of recombinant human biglycan (rhBGN) reduced dystrophic pathology and improved muscle function in mice with the same mutation that affects DMD boys.
The DMD Specialty Pharmacy Provider Directory is available at http://deltamarketingdynamics.
The objective of the collaboration is to reinforce existing preclinical proof of concept data in additional DMD animal models.
The fund is named after Harrison - the son of UK-based Alex and Donna Smith - who was diagnosed with DMD in January 2011.
DMD is caused by a mutation in the dystrophin gene, resulting in the absence or reduction of the dystrophin protein.
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