Raredr

Emerging Therapies for Duchenne Muscular Dystrophy

Jean Mah, MD
Published Online: Friday, Nov 04, 2016



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.

Latest Articles
Dr Michael Davidson of the University of Chicago School of Medicine discusses why a proper diagnosis is so valuable to FCS patients.
CRPS is often difficult to diagnose and in Jones’ case, that was also true. His symptoms appeared suddenly while playing high school football.
At the 2017 NLA Sessions, Rare Disease Report sat with dietician Wahida Karmally from Columbia University to discuss the necessary diet for FCS patients.
Michael Weschler, MD presents data at the 2017 American Thoracic Society Conference that shows the FDA-approved mepolizumab helps in the treatment of EGPA.
$vacMongoViewPlus$ $vAR$