Rare Disease Report
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Emerging Therapies for Duchenne Muscular Dystrophy

NOVEMBER 04, 2016
Jean Mah, MD



Jean L Mah, MD,  Associate Professor of Pediatric Neurology at Alberta Children's Hospital talks about the history, pathogenesis, treatment, and standard of care for boys with Duchenne muscular dystrophy. This symposium was filmed at the Child Neurology Society annual meeting in Vancouver, Canada on October 28, 2016.
 
Some of the therapies/targets discussed by Dr Mah include eteplirsen, SRP-053, NS-065, SRP-4045, DS-514b, ataluren, follistatin, AAV microdystrophin, GALGT2, myoblast transplant, cardiac stell cells, iPS stem cells, ezutrmid, domoagrozumab, BMS-986089, FG-3019, CAT-1004, givinostat, vamorolone, rimiporide, ARM210, nebivolol, spriromolactone, eplerenone, Ramipril, carvedilol, and idebenone. 
 
For more information about some of the clinical trials and therapies discussed by Dr Mah, visit jettfoundation.org.

Rare Disease Report would like to thank Dr Mah and the Jett Foundation for their support in allowing us to film this symposium and to share it with the rare disease community.

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