We all understand that Duchenne muscular dystrophy is characterized by muscle wasting and associated loss of function and that there are considerable efforts underway to develop drugs and biologics (cell and gene therapy) to address the primary problem in Duchenne—the absence of dystrophin. Restoring dystrophin or replacing dystrophin with replacement…Continue
Added by PPMD Partner Information on December 7, 2015 at 10:00am — No Comments
Come and advocate for Duchenne directly with members of Congress.
PPMD's Annual Advocacy Conference, held in the first quarter of each year, is the only officially organized gathering on Capitol Hill for the Duchenne community. Families…Continue
Added by Ryan Fischer on December 3, 2015 at 10:54am — No Comments