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All Blog Posts Tagged 'Staff' (310)


Staff
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


Staff
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…

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


Staff
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…

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


Staff
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 …

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


Staff
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…

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


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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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


Staff
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…

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


Staff
Strength Happens Together: PPMD Submits FDA Draft Guidance on Duchenne

June, 25, 2014 – Today is a landmark day for PPMD and the entire Duchenne community. We are so pleased that at our organization’s 20 year anniversary – after 2 decades of hard work and resolute persistence within the policy, legislative and regulatory arena – we are today able to provide to the US Food and Drug…

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Added by Pat Furlong on June 25, 2014 at 11:00am — No Comments


Staff
Illinois Designates Week of June 23 as Duchenne Awareness Week!

The State of Illinois is recognizing our 20th Annual Connect Conference in Chicago, IllinoisJune 26-29, 2014 by declaring the week of June 23 as Duchenne Muscular Dystrophy Awareness Week! With over 500 families and Duchenne experts expected…

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Added by PPMD on June 23, 2014 at 1:30pm — No Comments


Staff
PPMD Convenes Outcome Measures Meeting

PPMD will convene a pre-conference meeting of physical therapists, occupational therapists, and clinical coordinators who specialize in the research, assessment, longitudinal data collection, and treatment of people with Duchenne, June 25 and 26 in Chicago. This meeting will precede…

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Added by PPMD on June 19, 2014 at 2:30pm — No Comments


Staff
Progress Update! House Subcommitee Clears MD-CARE Act for Next Step

UPDATE - June 20, 2014

Progress! Yesterday the House Energy & Commerce Subcommittee on Health voted via unanimous voice vote to advance H.R. 594, the Paul D. Wellstone Muscular Dystrophy Community Assistance,…

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Added by Ryan Fischer on June 18, 2014 at 9:30am — No Comments


Staff
Parent Project Muscular Dystrophy Names Children's Hospital Colorado a Certified Duchenne Care Center

Children's Hospital Colorado (Children's Colorado) was named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to…

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Added by PPMD on June 17, 2014 at 12:32pm — No Comments


Staff
206th ENMC Workshop on Adults with Duchenne



We don’t know a lot about adults living with Duchenne: their natural history, cardiac progression on and off steroids, on an off cardiac medications, the effects of other medications and/or supplements, endocrine issues, gastrointestinal issues, urologic issues, gaining and maintaining independence……..the list could…

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Added by Kathi Kinnett on June 16, 2014 at 1:06am — No Comments


Staff
Pulmonary Care and Duchenne

Recently, Dr. Lisa Wolf (Assistant Professor, Pulmonary Medicine and Pulmonary and Neurology at Northwestern University, Feinberg School of Medicine, Chicago, IL) gave a wonderful presentation at Duchenne Foundation Australia’s Melbourne Symposium. While her presentation was titled, “DMD Respiratory Review 2014: The Younger Years,” she…

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Added by Kathi Kinnett on June 16, 2014 at 12:34am — No Comments


Staff
Healthy Summer Snacking & Activity



It’s summer – finally! Growing children are hungry children, and hungry children don’t usually go for the healthy snacks first.



As parents, we try everything we can to get them to eat right: reasoning (this sometimes works), bribery (generally referred to as “creative parenting”) and…

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Added by Kathi Kinnett on June 16, 2014 at 12:30am — No Comments


Staff
"Right to Try" State Model Legislation

The Goldwater Institute of Arizona has designed state model legislation, known as a "Right to Try" bill, which would make experimental drugs available to terminally-ill patients without the FDA's approval. Under this legislation, a patient with "an advanced stage of a disease with an unfavorable prognosis and no known…

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Added by PPMD on June 16, 2014 at 12:00am — 2 Comments

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