Leiden, The Netherlands – 29 January 2013 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, announced it has achieved orphan drug designation in the EU and the US for all of its compounds for the treatment of Duchenne muscular dystrophy (DMD). The European Medicines Agency […]

GSK Exon 51 Trial Update

Research
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GSK has sent out this update on their Duchenne trial.  The last patient has been recruited within the drisapersen US Phase II clinical study [www.clinicaltrials.gov NCT01462292]. This exploratory study aims to assess the safety, efficacy and pharmacokinetics of two doses of drisapersen in the treatment of ambulant boys with DMD who have a dystrophin gene […]
  Exon duplication mutations have been an area of great interest to CureDuchenne, and last year, CureDuchenne funded research specifically directed toward these mutations in Duchenne.  Dr. Kevin Flanigan at Nationwide Children’s Hospital has successfully completed the first phase of his research (please see below). CureDuchenne will continue to fund his work in 2013. The three […]
 We are so pleased to see the high caliber of professionals joining in the fight to cure Duchenne. Henri Termeer served as Chairman, President and CEO of Genzyme Corporation for nearly three decades.  He created a system where patients with rare diseases were able to have access to very expensive drugs. He joins an incredible […]
Drs. Carrie Miceli and Stan Nelson at UCLA are developing an FDA approved drug that could boost the effectiveness of exon skipping drugs that are being developed and tested for Duchenne muscular dystrophy. Drs. Miceli and Nelson in collaboration with Dr. Melissa Spencer, have tested dantrolene in combination with morpholinos in the mdx mouse, the mouse model […]
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 […]
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 […]
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 […]
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 […]
 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 […]
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