In June 2014, PPMD
drafted and submitted the first-ever patient advocacy-initiated guidance for a rare disease to the FDA to help accelerate development and review of potential therapies for Duchenne.
Following a change to Parent Project Muscular Dystrophy, the nonprofit opted for the more compact PPMD
is the sole guardian of DuchenneConnect and its material.
With the generous support of PPMD
, we are collaborating with the community to help maintain strength, enable a full range of motion, and improve the present and future for kids and adults with Duchenne.
has been an innovative leader in the emerging field of patient-focused drug development, organizing and convening the Duchenne community to create tools -- like the draft guidance document and with quantifiable benefit/risk preference data -- to be used by FDA to evaluate and approve treatments for boys with Duchenne who desperately await them," said Pat Furlong, Founder and President of PPMD
We are pleased that PPMD
has recognized Duke Children's Hospital & Health Center and our neuromuscular clinic as a leader in the diagnosis, care, and treatment of Duchenne muscular dystrophy," Dr.
We are honored that Ryan has chosen to make this generous gift to PPMD
In cooperation with the National Institutes of Health (NIH), PPMD
will announce the first-ever "End Duchenne Prize" - a one-year, $1 million grant awarded to a scientist working to advance translational research in Duchenne.
and Duchenne Parent Project NL have been early funders of several robotic programs that strive to improve the quality of life for people with Duchenne.
I am very grateful to PPMD
for providing funds at this critical time in our studies", says Dr.
's preeminent role in the Duchenne muscular dystrophy community, we are honored that the Stanford Comprehensive Neuromuscular Program is being recognized as a PPMD
Certified Duchenne Care Center," said Dr.
Last year, PPMD
initiated an innovative Xcelerate grant competition in recognition of the large number of exciting late stage preclinical and early stage clinical therapeutics in the pipeline for Duchenne.