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.

Sarepta Update

Sarepta provided…

Cincinnati Children’s Hospital Medical Center has been named the third Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD).

PPMD established certified…

The House of Representatives today approved the landmark MD-CARE Act so the law can continue getting results for all patients impacted by Duchenne and other forms of muscular dystrophy. The House approved the bill, H.R. 594, sponsored by Congressman Dr. Michael Burgess of Texas and Eliot Engel of New York, by unanimous voice vote. …

MD-CARE Act Clears Senate HELP Committee 

Today, the Senate HELP Committee approved the MD-CARE Act - another huge step in the right direction for our bill. This…

Please take 1 minute to email your Senators!

Today, a key Senate committee will …

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…

Everyone loves a clever sales pitch. And coming up with something simple, short, and clever is the best way for you to turn your Duchenne fundraising idea into a successful reality! Sometimes just a simple lemonade stand, with a great tagline can change hearts and raise money.

Thanks to the "PPMD Lemonade Divas" pictured above for…

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 Deutekom, parents wanted to know what it is we are actually funding.…

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…

Please take 1 minute to email your Senators!

Yesterday, a key Senate committee announced that it will …

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 use of patient reported outcomes in drug…

Care of the heart in Duchenne is not a simple business. As a community, we realize:  

• Prescriptions for cardiac care and surveillance differ widely between both practices and providers.
• Large gaps exist in cardiac research.
• While future goals are necessary, we need a plan, for both cardiac care and research, and we need it…

One Step Closer

Progress! Today the House Energy & Commerce committee voted unanimously to advance H.R. 594, the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2014. This action moves the bill to consideration and hopefully passage by the full…

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 adult cardiologists as well as clinical researchers, will meet…

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 says, “go!” or when the child takes the first step? If there’s a “false start” do…

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 focuses on overcoming challenges in clinical trial design across…

Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD and mom to Hazel, age 8, and Rain, age 6. Rain has Duchenne muscular dystrophy. Follow Ivy at her blog, Living …

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 …

If you are always trying to be normal, you will never know how amazing you can be.

― Maya Angelou

What do you want to be when you grow up? The question Dr. Sue Apkon asks every boy with Duchenne (girls too). The question, that for so many years brought tears to our eyes and a lump in our throat. The question that always resulted in mental math... if this happens and that happens, then maybe…. But now, it is reality. Our sons…