PPMD Community

All Blog Posts Tagged 'Staff' (339)


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…

Continue

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…

Continue

Added by PPMD on October 23, 2014 at 9:00am — No Comments


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…

Continue

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…

Continue

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…

Continue

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…

Continue

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…

Continue

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…

Continue

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…

Continue

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…

Continue

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 …

Continue

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


Staff
Congressional 21st Century Cures Initiative: PPMD Ensuring our Duchenne Community’s Place At the Table

PPMD is continuously seeking innovative opportunities to impact the process of developing therapies for Duchenne. Opportunity comes in many forms - funding promising research, advancing care standards for all patients, and advocating for key changes in federal policy.

 

Earlier this year Congress launched the 21st Century Cures Initiative, a comprehensive look at what can be done to accelerate the pace of cures in American therapeutic…

Continue

Added by Ryan Fischer on September 15, 2014 at 12:00pm — No Comments


Staff
Social Security Disability Help

If you have a child with Duchenne or Becker muscular dystrophy, you may be eligible to receive funds from the government to help with the burden of expenses brought on by day to day life with muscular dystrophy. Social Security Disability benefits, in the form of Supplemental…

Continue

Added by Brian Denger on September 15, 2014 at 11:30am — No Comments


Staff
Susie’s Story

In 2013, we brought you the story of Jason and Susie, two courageous patients who had undergone implantation of left ventricular assist devices (LVADs).…

Continue

Added by Kathi Kinnett on September 15, 2014 at 10:54am — No Comments


Staff
It's Flu Season Again!

Note: Several of you have also asked about the enterovirus, a mild to severe respiratory illness going around now. The CDC has provided helpful…

Continue

Added by Kathi Kinnett on September 15, 2014 at 10:30am — No Comments


Staff
We Got Annie

Timing is everything. When I think back on how I found Pat, Kimberly, and PPMD ten years ago to the month, I often think about the events that led to that faithful interview in the fall of September 2004. Sure, maybe some of it was luck, but I also believe…

Continue

Added by Ryan Fischer on September 10, 2014 at 4:00pm — No Comments


Staff
‘Operation Duchenne’: Proud to Be Serving In The Trenches With You

Each member of our Duchenne community has a personal reason for being here. Much like soldiers in an army, some of us have been drafted while others have us have enlisted – but we are all fighting together, battle after battle - within the same trenches.…

Continue

Added by Annie Kennedy on September 9, 2014 at 12:00pm — 1 Comment


Staff
Action Alert: Tell the Senate to take action on MD-CARE Act!



Congress has returned from their Summer recess and it is time to re-engage them on the MD-CARE Act Amendments. We need each member of our community to reach out to your Senators and urge them to tell leadership to bring the…

Continue

Added by Ryan Fischer on September 9, 2014 at 10:35am — No Comments


Staff
Let’s improve DuchenneConnect’s informed consent. Give your opinion by September 7th.

You may be thinking, “An informed consent document? No way do I want to read one of those!” But you can demonstrate the power of our community and help us write a more understandable, responsible informed consent for our community’s registry.



As many of you know,…

Continue

Added by Holly Peay on August 28, 2014 at 11:25am — No Comments

Monthly Archives

2014

2013

2012

2011

2010

2009

2008

2007

© 2014   Created by PPMD.

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service