PPMD's Blog – July 2014 Archive (9)

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…

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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…

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Added by PPMD on July 29, 2014 at 11:30am — 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…

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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…

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Added by PPMD on July 22, 2014 at 12:58pm — 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…

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Added by PPMD on July 16, 2014 at 9:30am — 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…

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

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

Independence

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 …

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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 …

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

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