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A preclinical study led by researchers at Children’s National Medical Center has found that a new oral drug shows early promise for the treatment of Duchenne muscular dystrophy (DMD).
This is progress on the ReveraGen drug, a new chemical entity that has been developed as a potential steroid replacement, without the significant side effects of steroids. This is amazing progress and we are hopeful/anxious to see this move into clinical studies.
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As researchers embark on a project to connect 30 million patient records, questions about privacy arise.
Commentary by Eric Hoffman and Beth McNally.
MILFORD, Mass. and RENO, Nev. , Feb
We are heartbroken to learn more discouraging news about another Duchenne therapy today, HT-100 from Halo Therapeutics. This community continues to face tremen…
PTC Therapeutics, Inc., today announced the publication of data in PLOS ONE demonstrating that nonsense mutation Duchenne muscular dystrophy (nmDMD) patients treated with ataluren, an investigational new drug, experienced an increase in dystrophin expression. These data were obtained from PTC's Phase 2a open-label trial of ataluren in which change in full-length dystrophin expression, as assessed by immunofluorescent staining, was the primary endpoint.
Proactive Investors UK Summit Corp, Oxford University in Duchenne alliance Stock Market Wire StockMarketWire.com - Summit Corp, a drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy (DMD) and C.
PTC Therapeutics, Inc., a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small molecule drugs that target post-transcriptional control processes, today announced financial and corporate results for the quarter ended September 30, 2013.
Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced the successful enrollment of the 100th patient into the Natural History Study of Duchenne muscular dystrophy (DMD).
The goal of this observational study is to characterize DMD at various stages of progression using the same measures used in ongoing clinical studies, such as the "six minute walk test." No medication is being tested in this study.
Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, today announced that Michael S. Wyzga has been nominated for appointment to its Supervisory Board at the next shareholders' meeting, scheduled for June 17, 2014.
Prosensa just released their 48-week data for drisapersen, as well as an update on recent corporate developments. Both press releases are included below. PPMD…
Prosensa just released their 48-week data for drisapersen, as well as an update on recent corporate developments. Both press releases are included.
PPMD has been talking with Prosensa and will be hosting a webinar with UPPMD later this month. We will be asking you for questions to pass along to Prosensa prior to the webinar. More to come soon.
When treated with nanoparticles loaded with an immunosuppressive drug, mice with muscular dystrophy showed improved strength and heart function.
Analyses and assessment of next steps are ongoing PPMD just received the following update from GSK regarding the current status of their drisapersen study: D…