This report on treatment development for Duchenne muscular dystrophy contains news about fighting fibrosis (scarring), treating DMD-related heart disease, exon skipping and the corticosteroid deflazacort
posted on February 2, 2015 - 12:04pm
Development of treatments for Duchenne muscular dystrophy (DMD) continues to advance. Many of the new investigational drugs are potentially applicable to all DMD patients, while a few target those with specific mutations in the dystrophin gene.
Becca Niebrugge has long been inspired by her uncle Mike Mitchell, who, like Becca’s dad, was an avid long-distance runner. But it was the painful experience of watching ALS (amyotrophic lateral sclerosis) abruptly strip her uncle of his ability to run, before ultimately claiming his life in April 2013, that finally compelled Becca to tackle a full marathon herself.