July 2014 Blog Posts (19)

Exon-Skipping Trial Updates from Sarepta & Prosensa

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…


Added by PPMD on July 31, 2014 at 3:00pm — No Comments

Cincinnati Children’s Named Certified Duchenne Care Center By Parent Project Muscular Dystrophy

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


PPMD established certified…


Added by PPMD on July 29, 2014 at 11:30am — No Comments

BREAKING NEWS: Houses of Representatives Passes MD-CARE Act Amendments!

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. …


Added by Pat Furlong on July 28, 2014 at 6:12pm — No Comments

Senate HELP Committee Approves MD-CARE Act - Another Critical Step Forward for our Bill

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…


Added by Ryan Fischer on July 23, 2014 at 1:00pm — No Comments

Action Alert! Senate HELP Committee Marks Up MD-CARE Act Today -Take a Moment to Contact your Senators

Please take 1 minute to email your Senators!


Today, a key Senate committee will …


Added by Ryan Fischer on July 23, 2014 at 11:00am — No Comments

Navigating the Landscape of Compassionate Use

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…


Added by PPMD on July 23, 2014 at 9:13am — No Comments

Spotlight: PPMD Lemonade Divas

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…


Added by PPMD on July 22, 2014 at 12:58pm — No Comments

No Exon Left Behind

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.…


Added by Sharon Hesterlee on July 21, 2014 at 10:15am — 6 Comments

NIH Awards $6.3 Million Grant to Seattle Muscular Dystrophy Researchers

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 CenterCongratulations to Dr. Chamberlain and…


Added by Ryan Fischer on July 18, 2014 at 11:28am — No Comments

Action Alert! Key Senate Committee Schedules Markup for MD-CARE Act

Please take 1 minute to email your Senators!


Yesterday, a key Senate committee announced that it will …


Added by Ryan Fischer on July 17, 2014 at 12:30pm — No Comments

PPMD President Participates on Patient Reported Outcomes Panel to Inform FDA Public Meeting

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…


Added by PPMD on July 16, 2014 at 9:30am — No Comments

Duchenne Cardiac Working Group Recap

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…

Added by Kathi Kinnett on July 15, 2014 at 2:30pm — No Comments

Energy and Commerce Committee Clears MD-CARE Act for House Vote

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…


Added by Ryan Fischer on July 15, 2014 at 10:30am — No Comments

PPMD and NHLBI Co-Sponsor Duchenne Cardiac Working Group

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…


Added by PPMD on July 9, 2014 at 10:02am — 3 Comments

Outcomes Meeting: Laying a Foundation for Outcome Measures

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”…


Added by Kathi Kinnett on July 8, 2014 at 11:30am — No Comments

FDA Draft Guidance on Duchenne: Telebriefing

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…


Added by PPMD on July 7, 2014 at 11:30am — No Comments


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 …


Added by PPMD on July 3, 2014 at 7:03pm — 1 Comment

International Study on the Burden of Duchenne Published in Neurology

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 …


Added by PPMD on July 3, 2014 at 12:00pm — No Comments

Conference 20

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…


Added by Pat Furlong on July 2, 2014 at 11:00am — No Comments

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