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nNOS: How Important is it in Duchenne?
nNOS has been in the news lately so we asked CureDuchenne’s chief scientific advisor, Dr. Mike Kelly to answer a few questions. Question: what is nNOS and why do we need it? nNOS […]
CureDuchenne – Clinical Trials – FDA
Following the long awaited news of Sarepta’s phase IIB results for eteplirsen and continued Phase III progress of GSK/Prosensa’s drisapersen, as well as the recent developments with PTC Therapeutics and […]
Tadalafil alleviates muscle ischemia in patients with Becker muscular dystrophy
A recent placebo-controlled clinical study on the effects of the PDE5A inhibitor drug Tadalafil (Cialis), demonstrated beneficial effects on muscle blood flow in a model of muscle ischemia in men with Becker muscular dystrophy who harbor a mutation in which the neuronal nitric oxide synthase is absent. The result reinforce earlier findings in mice and Duchene patients and highlight the role of this pathway in muscle homeostasis.
Sarepta Therapeutics Enters Into Collaboration for the Development of Additional Exon-Skipping Product for Duchenne Muscular Dystrophy
November 26, 2012 08:30 ET Collaboration Represents Significant Progress in Sarepta’s Path to Develop Treatments for Broader DMD Population CAMBRIDGE, MA–(Marketwire – Nov 26, 2012) – Sarepta Therapeutics (NASDAQ: SRPT), […]
Sarepta Therapeutics Enters Into Collaboration for the Development of Additional Exon-Skipping Product for Duchenne Muscular Dystrophy
November 26, 2012 08:30 ET Collaboration Represents Significant Progress in Sarepta’s Path to Develop Treatments for Broader DMD Population CAMBRIDGE, MA–(Marketwire – Nov 26, 2012) – Sarepta Therapeutics (NASDAQ: SRPT), […]
REPEAT DOSING OF SMT C1100 FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY MEETS ENDPOINTS IN PHASE 1 CLINICAL TRIAL
New formulation delivers drug levels that are predicted to significantly increase utrophin production Summit to progress utrophin upregulator into next stages of development Oxford, UK, 7 November 2012 – Summit […]
Duchenne Muscular Dystrophy is Closer to a Treatment with New Results from Sarepta Therapeutics, Cure Duchenne Early Supporter of Study
Newport Beach, Calif., October 3, 2012: CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, is pleased by today’s announcement by Sarepta […]
CureDuchenne Founder to Participate in Global Genes RARE Project Tribute to Champions Event, RARE Advocacy Summit and Documentary Screening
Newport Beach, Calif., SEPTEMBER 27, 2012: CureDuchenne Founder, Debra Miller, will speak at the Global Genes | R.A.R.E. Project “Tribute to Champions of Hope” benefit and RARE Patient Advocacy Summit […]
Anaheim Ducks Captain Ryan Getzlaf to Host 2nd Annual Getzlaf Golf Shootout This Weekend To Benefit Cure Duchenne
Newport Beach, Calif., SEPTEMBER 5, 2012: Ryan Getzlaf, captain of the Anaheim Ducks, is hosting the second annual Getzlaf Golf Shootout on September 9 to benefit CureDuchenne, a nonprofit that […]