1. Cure Duchenne Supports Duchenne Duplication Mutation Research with Dr. Kevin Flanigan

    Posted on December 28, 20126:42 pm

      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 Read more »

  2. Henri Termeer joins Prosensa as Strategic Advisor

    Posted on December 13, 20125:01 pm

     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 Read more »

  3. Dantrolene: An Exon Skipping Enhancing Drug

    Posted on December 12, 20128:52 pm

    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 Read more »

  4. nNOS: How Important is it in Duchenne?

    Posted on December 11, 201210:58 pm

    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 »

  5. CureDuchenne – Clinical Trials – FDA

    Posted on December 7, 201212:45 am

    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 »