You may be thinking, “An informed consent document? No way do I want to read one of those!” But you can demonstrate the power of our community and help us write a more understandable, responsible informed consent for our community’s registry.
As many of you know,…Continue
Added by Holly Peay on August 28, 2014 at 11:25am — No Comments
Two NEW and exciting races have been added to our Run For Our Sons calendar for 2015....which adventure will you…
Added by Nicole Herring on August 19, 2014 at 1:30pm — No Comments
As you know, PPMD recently facilitated a multi-stakeholder process involving patients, parents, investigators, industry, and other interested parties which culminated in a draft guidance document…Continue
Added by Pat Furlong on August 18, 2014 at 1:00pm — No Comments
Sure, we’re in the lazy days of summer and Congress is on recess, but now is not the time to slow down on the MD-CARE Act. Let’s keep our momentum going. Take a minute to thank the House for passing the…Continue
Added by Ryan Fischer on August 13, 2014 at 9:30am — No Comments
The lungs require the strength of many muscles to help a person to breathe and cough. As people with Duchenne grow older, the respiratory muscles, like the skeletal muscles, become weaker.…Continue
Added by Kathi Kinnett on August 12, 2014 at 9:30am — No Comments
Every year I try to escape the real world for a couple of days and retreat with my family to probably my favorite place on Earth – Dowagiac. I’ve reminisced before, but this place is magical to me. It is my childhood, it is summer, it is magic. With so much going on in our community –…Continue
We are excited to welcome the two newest members of our Scientific Advisory Committee, Dongsheng Duan, PhD and Jill Rafael-Fortney, PhD! Our Scientific Advisory Committee, chaired by PPMD Senior Scientific Advisor Dr. Lee Sweeney, includes leading experts in basic and clinical Duchenne research and Duchenne clinical management. The committee expertly reviews every grant that comes in and offers funding or advice on…Continue
Added by PPMD on August 6, 2014 at 11:30am — No Comments
I had no words for Scott’s death. In fact, he had been actively dying for several years. I had hoped that he might see Rebekah for one last time, though I realized they were both too sick and living in different countries made it impossible. The good news is that many people with Duchenne never find…Continue
Added by Pat Furlong on August 4, 2014 at 10:30am — No Comments
In the wake of promising exon-skipping trial announcements earlier this summer, PPMD has been fielding many questions from the community about upcoming trials. We reached out to both Sarepta and Prosensa for updates to share.…
Added by PPMD on July 31, 2014 at 3:00pm — No Comments
Cincinnati Children’s Hospital Medical Center has been named the third Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD).
PPMD established certified…Continue
Added by PPMD on July 29, 2014 at 11:30am — No Comments
Requests for compassionate use of investigational drugs have increased dramatically in recent years.
In a newly released white paper by Siren Interactive, produced from input…Continue
Added by PPMD on July 23, 2014 at 9:13am — No Comments
I'm often asked by parents to explain exon skipping. In fact, at last month's 20th Annual Connect Conference, after we announced a grant awarded to Dr. Judith van…Continue
A group of Seattle-based muscular dystrophy researchers has been awarded a prestigious $6.3 million grant from the National Institute of Health (NIH) to establish a Senator Paul D. Wellstone Cooperative Research Center. Congratulations to Dr. Chamberlain and…
Added by Ryan Fischer on July 18, 2014 at 11:28am — No Comments
Please take 1 minute to email your Senators!
Yesterday, a key Senate committee announced that it will …Continue
Added by Ryan Fischer on July 17, 2014 at 12:30pm — No Comments
Today, PPMD President Pat Furlong has the distinct honor of participating on a panel at the Engelberg Center for Health Care Reform at Brookings.
The topic will be: Enhancing the development of and…Continue
Added by PPMD on July 16, 2014 at 9:30am — No Comments
Care of the heart in Duchenne is not a simple business. As a community, we realize:
Added by Kathi Kinnett on July 15, 2014 at 2:30pm — No Comments
Parent Project Muscular Dystrophy (PPMD) has partnered with the National Heart, Lung, and Blood Institute (NHLBI) to convene a working group titled “Contemporary Cardiac Issues in Duchenne Muscular Dystrophy.” The group, made up of top pediatric and…Continue
A 6 minute walk test is a 6 minute walk test, right?
Well, maybe not.
It turns out, there can be many variations of the same test. For example, is it done on carpet? On linoleum? On concrete? Shoes on? Shoes off? Socks on? Socks off? Do you start when the therapist…Continue
Added by Kathi Kinnett on July 8, 2014 at 11:30am — No Comments
On June 25, 2014, we shared the first-ever rare disease patient-initiated guidance with FDA to help accelerate treatments for Duchenne. This new guidance…
Added by PPMD on July 7, 2014 at 11:30am — No Comments
In the first international study of its kind, researchers have found that there are many different costs accompanying a rare condition such as Duchenne muscular dystrophy and that there is a considerable financial burden carried by affected families. Funded by GlaxoSmithKline (GSK), the aim of this study was to estimate the total cost of illness and the economic burden of Duchenne.
The research, published today in the journal …Continue
Added by PPMD on July 3, 2014 at 12:00pm — No Comments