A Note from Cure Duchenne Founder, Debra Miller

Today BioMarin announced they will initiate a human trial for a drug that will hopefully up-regulate Utrophin.  This is very exciting news since we have hope that Utrophin could be an adequate substitute for dystrophin, the protein that is lacking in patients with Duchenne muscular dystrophy (DMD).  This is the very first Utrophin drug to make it to human clinical trials – We applaud  Dr. Kay Davies for developing this drug, to BioMarin for investing in this drug, and for Action Duchenne for helping fund it along the way.

My hope is that next year at this time, we will have several new drugs in the trial process for DMD and that the ones already in trial, prove to be effective and safe.  The funding for the early development of these drugs is crucial and I’d like to encourage anyone who has an interest in curing DMD to become active in raising funds for research. 

Of course, we’d love to have you join us in the many fundraising opportunities that CureDuchenne provides, such as our upcoming mountain climb and bike relay.  These will be announced shortly.

Debra Miller

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California Healthcare Institute publishes Interview with Cure Duchenne Founder Debra Miller

California Healthcare Institute (CHI) recently published an interview with Cure Duchenne Founder Debra Miller. In this very informative interview, Debra discusses her personal history with Duchenne Muscular Dystrophy, the DMD culture in general, the possibility of treatments, and the need for funding to find a cure.  

As stated by CHI:

CHI will launch three new podcasts focused on rare diseases, jointly produced by CHI, The Burrill Report, and the Children’s Rare Disease Network. In the first podcast, on July 10, Debra Miller, president and founder of Cure Duchenne, describes her experiences when she faced the grim news that her five-year-old son was diagnosed with Duchenne Muscular Dystrophy. The progressive muscle loss caused by the disease typically leaves these children wheelchair-bound by age 10; few survive beyond their twentieth year. As she and her husband explored treatment options and ways they could raise money for research, they found that existing organizations were often focused on palliative care and pursuing academic research rather than working with industry. Miller and her husband launched Cure Duchenne to help fund promising research at biotech companies in the hopes of seeing these therapies developed and commercialized. We spoke to Miller about her son’s story, new approaches to treating Duchenne, and the problems of funding cutting-edge research.

To listen to the Podcast, just press the blue play button (or you can also right click the text link and choose “save target as” or “save link”)

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