11 Dec
  • By cure.duchenne
  • Cause in

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 stands for neuronal nitric oxide synthase; its function is to produce nitric oxide and when needed, (in a simplistic view) help increase blood flow in […]

READ MORE
07 Dec
  • By cure.duchenne
  • Cause in

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 Summit PLC among others, CureDuchenne took the initiative to reach out for patients and advocacy groups alike to begin a dialogue with the FDA and […]

READ MORE
26 Nov
  • By cure.duchenne
  • Cause in

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), a developer of innovative RNA-based therapeutics, announced today a collaboration for the development of an additional exon-skipping drug targeting exon 53, its fourth drug in […]

READ MORE
26 Nov
  • By cure.duchenne
  • Cause in

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), a developer of innovative RNA-based therapeutics, announced today a collaboration for the development of an additional exon-skipping drug targeting exon 53, its fourth drug in […]

READ MORE
07 Nov
  • By cure.duchenne
  • Cause in

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 (AIM: SUMM), a UK drug discovery company, announces that the repeat dosing of the utrophin upregulator SMT C1100 for the treatment of the fatal muscle-wasting […]

READ MORE