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All Blog Posts Tagged 'Staff' (347)


Staff
Help us bring the ABLE Act to the Floor – it’s a game changer!

Great news... House and Senate Leadership continue to hear from the disability community that the Achieving a Better Life Experience (ABLE) Act (S. 313/H.R. 647) must pass this year!



The ABLE Act amends Section 529 of the U.S. Tax Code that…

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Added by Annie Kennedy on November 21, 2014 at 9:00am — No Comments


Staff
How can we best advance the science of patient input to improve the efficiency of the drug development and regulatory review process?



PPMD and other leaders in the rare disease community were asked by…

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Added by Ryan Fischer on November 20, 2014 at 11:30am — No Comments


Staff
PPMD Names Baltimore’s Kennedy Krieger Institute a Certified Duchenne Care Center

Kennedy Krieger Institute in Baltimore, Maryland was named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working…

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Added by PPMD on November 18, 2014 at 1:28pm — No Comments


Staff
Pfizer Myostatin Blocker Trial Set to Launch

At the Action Duchenne meeting in London on Saturday, Pfizer Inc. announced that it has completed a study in healthy volunteers with its antibody-based therapeutic to block the muscle protein myostatin, and is preparing to start recruitment for a phase II study in Duchenne this month. Watch…

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Added by PPMD on November 14, 2014 at 12:59pm — No Comments


Staff
Duchenne Pulmonary Care for Tweens and Teens (Webinar Summary & Recording)

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. Like the heart, every person with Duchenne will have pulmonary issues of some…

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Added by Kathi Kinnett on November 6, 2014 at 10:00am — No Comments


Staff
Patients Are Waiting...

The FDA is listening and has told us as much. But that doesn’t mean we should stop doing everything we can to educate the Agency.



Thanks to the dozens of you who participated in PPMD’s benefit/risk project and took the time to share your story, we have been able to compile your responses and…

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Added by PPMD on November 3, 2014 at 7:30am — No Comments


Staff
PPMD Submits Letter to FDA Regarding Latest Sarepta Update

Today’s press release and investor call from Sarepta have sent a ripple of angst and sadness through our community. Moments ago, PPMD sent a letter to the FDA that reflects our organization’s belief that safety and rigor have been demonstrated throughout this process. Further, we…

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Added by PPMD on October 27, 2014 at 4:23pm — 2 Comments


Staff
Enterovirus

Everyone is well aware that we are in the thick of flu season. Recently, PPMD board member, and Duchenne parent, Ellen Wagner, got first-hand experience dealing with the scariness of the…

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Added by Kathi Kinnett on October 25, 2014 at 11:30am — No Comments


Staff
Notes from FDA’s Presentation at NORD Summit

Dr. Janet Woodcock, Director of FDA's Center for Drug Evaluation and Research (CDER) presented the opening keynote at NORD's Rare Diseases and Orphan Products Breakthrough Summit in Washington, DC on October 21.



Her remarks focused on how the patient advocacy community can most effectively…

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Added by Ryan Fischer on October 23, 2014 at 9:30am — No Comments


Staff
PPMD-Funded Gene Therapy Study Improves Walking Ability in Muscular Dystrophy

Dr. Jerry Mendell of Nationwide Children’s Research Institute recently presented data from a ground-breaking PPMD-funded study demonstrating that a modified virus carrying the gene for follistatin can improve performance on the 6 minute wal…

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Added by PPMD on October 23, 2014 at 9:00am — 1 Comment


Staff
News from World Muscle Society: Day 4

Late Breaking Talks:

PPMD-funded gene therapy study improves walking ability in muscular dystrophy

Dr. Jerry Mendell of Nationwide Children’s Research Institute presented data from a ground-breaking…

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Added by PPMD on October 13, 2014 at 9:30am — No Comments


Staff
News from World Muscle Society: Days 2 & 3

PTC Therapeutics Symposium – “Improving Outcomes for the Future: Making sense of the nonsense mutations in Duchenne muscular dystrophy”

PTC Therapeutics gave an overview of the data supporting the recent accelerated approval…

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Added by PPMD on October 10, 2014 at 9:40am — No Comments


Staff
News from World Muscle Society Meeting (10/8/14)

Sarepta held a symposium on the role of dystrophin production in disease-modifying treatments for Duchenne. Some highlights:

 

We heard from Dr. Francesco Muntoni from the Dubowitz Neuromuscular Centre about how much…

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Added by PPMD on October 9, 2014 at 9:00am — No Comments


Staff
Calling All Adults with Duchenne/Becker – This Means YOU!

Ever wished you could change the world – or at least your piece of it? Been looking for a way to engage a bit more? Have personal experience that you’d like to see be better reflected in policy and program efforts?

PPMD is seeking Adult members of our Duchenne/Becker community to serve on the…

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Added by Annie Kennedy on October 6, 2014 at 10:14am — No Comments


Staff
Early Pulmonary Care Webinar Summary & Recording - 9/24/2014

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. Like the heart, every person with Duchenne will have pulmonary issues of some…

Continue

Added by Kathi Kinnett on October 3, 2014 at 9:00am — No Comments


Staff
President Signs MD-CARE Act Amendments Into Law

Capping a two-year advocacy campaign led by Parent Project Muscular Dystrophy (PPMD), today President Barack Obama signed the MD-CARE Act…

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Added by PPMD on September 26, 2014 at 4:30pm — No Comments


Staff
The MD-CARE Act: Reflections & Projections

With the passage of the Amendments to the MD-CARE Act last week, there is unprecedented momentum for Duchenne at the federal level. And with this major victory, words and phrases echo from the…

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Added by Pat Furlong on September 23, 2014 at 12:30pm — 1 Comment


Staff
ALD. Ever heard of it?

No, not the ‘Ice bucket thing’. That’s ALS. This is Adrenoleukodystrophy (ALD). It is an X-linked recessive disorder that affects an estimated 1 in every 18,000 live births. It is primarily a childhood disorder where boys are born appearing completely healthy until symptom unset sometime…

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Added by Annie Kennedy on September 22, 2014 at 11:30am — No Comments


Staff
MD-CARE Act Passed!

Duchenne Community Knows No Bounds

Today – September 18, 2014, our Duchenne community once again made history by demonstrating that nothing could stop us from ensuring that the life-saving research momentum of our landmark bill would be expanded to reflect the current needs of our…

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Added by Pat Furlong on September 18, 2014 at 10:13pm — 2 Comments


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

Fourth Clinic to Receive Certification

Seattle Children's Hospital was named a Certified Duchenne Care Center by …

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Added by PPMD on September 17, 2014 at 3:59pm — No Comments

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