TREAT-NMD Presents Annemieke Aartsma-Rus’s Overview of DMD Research

Please take some to head over to an important article on Treat-NMD’s website to learn about Annemieke Aartsma-Rus’s overview of Duchenne muscular dystrophy (DMD) research:

TREAT-NMD presents Annemieke Aartsma-Rus’s overview of DMD research

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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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Diagnosis and Management of Duchenne Muscular Dystrophy

Please take some time to read this important document regarding the diagnosis and management of Duchenne Muscular Dystrophy (DMD). As the document itself states, “although guidelines are available for various aspects of DMD, comprehensive clinical care recommendations do not exist. The US Centers for Disease Control and Prevention selected 84 clinicians to develop care recommendations using the RAND Corporation–University of California Los Angeles Appropriateness Method.

These recommendations, presented in two parts, are intended for the wide range of practitioners who care for individuals with DMD.”

Click here to read:

Diagnosis and Management of Duchenne Muscular Dystrophy

 

 

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