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Annie Kennedy's Blog (49)

At Long Last, PDUFA VI Hearings Have Begun – But There’s a Twist

Wow...what a week in Washington! President Trump released his budget proposal, the American Health Care Act moves to the floor of the House for a vote today, the U.S. Supreme Court ruled on a landmark case around the rights of students with disabilities within the public school system, and at long last after months of our Duchenne…

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Added by Annie Kennedy on March 23, 2017 at 10:30am — No Comments

ACTION ALERT: Make Your Voice Heard on New Healthcare Proposal

“Who’s on first?”

Remember that classic Abbott & Costello routine? Well, trying to track the quickly-shifting healthcare bill has felt much like watching that routine. Complex, confusing, and exhausting. But the stakes here are no laughing matter.

 

We…

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Added by Annie Kennedy on March 21, 2017 at 10:00am — No Comments

PPMD Joins Rare Disease Advocates in Letter to President

PPMD joined 200 patient organizations in a letter to President…

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Added by Annie Kennedy on February 27, 2017 at 7:30pm — No Comments

Meet the 2017 PPMD Adult Advisory Committee (PAAC)

Years ago, a friend of mine named Tom Bailey who had Duchenne wrote a memoir that he entitled, Moving Mountains Without Muscles. As I look at the tremendous accomplishments of PPMD’s Adult Advisory Committee since we first launched this leadership group 2 years ago, I am astounded by how…

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Added by Annie Kennedy on January 16, 2017 at 7:30pm — No Comments

New Resource to Help Navigate EXONDYS 51 Access

Prior to the approval of EXONDYS 51™, we knew that access to emerging therapies would be a process – and an often challenging process at…

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Added by Annie Kennedy on January 13, 2017 at 11:00am — No Comments

BREAKING NEWS: 21st Century Cures Crossed Over the Finish Line!

The Duchenne Community’s Voice Was Essential Every Step of the Way & Contains PPMD’s Provision to Strengthen Patient Focused Drug Development Efforts



To everyone in our PPMD community who called, reached out, emailed, and met with your elected…

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Added by Annie Kennedy on December 7, 2016 at 2:30pm — No Comments

Understanding Insurance Determinations



With the accelerated approval of EXONDYS 51™ (aka eteplirsen) by the FDA in September, the Duchenne community has been quickly…

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Added by Annie Kennedy on November 2, 2016 at 12:30pm — No Comments

The Assistance Fund Launches Duchenne Program to Support Access to Exondys 51

Patient Assistance programs are typically non-profit organizations sponsored by drug companies, patient advocacy organizations, and civic groups that establish disease specific programs to facilitate access to brand-name medications.



In the case of…

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Added by Annie Kennedy on October 28, 2016 at 11:00am — No Comments

ABLE Accounts: Improvements Moving Through Congress & Determining Which State’s Program is Best for You?

With the initiation of 4 states ABLE programs already begun and Oregon having just anounced that their program will launch in December, any qualified individual (meaning most families with Duchenne) can open their accounts in 2016, if interested. We have previously shared information…

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Added by Annie Kennedy on October 13, 2016 at 8:37pm — No Comments

Calling All Adults with Duchenne/Becker – Join the 2017 PPMD Adult Advisory Committee!

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 national policy and program efforts?



PPMD is seeking adult members of our Duchenne/Becker…

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Added by Annie Kennedy on October 12, 2016 at 11:30am — No Comments

[Webinar Takeaways] Preparing for Access to Approved Therapies

Last week, PPMD began a webinar series on access – an exciting new chapter for our Duchenne community. But we are just at the beginning of this chapter and there is lots to explore.



We knew this day was coming –…

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Added by Annie Kennedy on October 11, 2016 at 3:00pm — No Comments

September is National Newborn Screening Awareness Month



September is National Newborn Screening Awareness month and PPMD has been helping to put Duchenne on the newborn screening map — the WORLD map.

Our community understands the inherent…

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Added by Annie Kennedy on September 14, 2016 at 11:30am — No Comments

PPMD Continues to Lead Work on Critical Duchenne Access Issues

With the promise of drug approvals on the horizon, access to these therapies will be critical to our Duchenne community. PPMD has been leading work related to Duchenne and rare disease community access issues on multiple fronts. …

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Added by Annie Kennedy on September 14, 2016 at 11:25am — No Comments

The Importance of Early Diagnosis: Eliminating the Diagnostic Odyssey

The global Duchenne community will come together on September 7 for World Duchenne Awareness Day. This year a theme was chosen that is not…

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Added by Annie Kennedy on September 1, 2016 at 1:30pm — No Comments

Essential Back-To-School Resources for Duchenne

I have a confession…

 

I have had the privilege of serving as an advocate for families whose children have Duchenne within school systems for more than 20 years, have attended hundreds of IEPs and 504 Plan meetings, and have given even more school presentations, assemblies and…

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Added by Annie Kennedy on August 29, 2016 at 1:30pm — No Comments

PDUFA VI & Its Impact on Duchenne

On Friday, PPMD submitted additional comments to the latest version of PDUFA VI.  At this point, there are few members of our Duchenne community who haven’t heard the acronyms ‘PDUFA’ or…

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Added by Annie Kennedy on August 19, 2016 at 1:38pm — No Comments

Understanding ABLE Programs & How You Can Benefit



We know that many within our community have long been advocating for and following the passage of The Achieving a Better Life Experience (ABLE) Act and subsequent IRS rules and state implementation. In June, the first state ABLE programs launched with the initiation of Ohio’s STABLE program. At …

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Added by Annie Kennedy on August 12, 2016 at 9:52am — No Comments

PPMD Responds to Threat to Lose DOD Funding

Our Duchenne community’s life-saving research efforts are funded through a number of public and private sources. Beginning in FY 2011, the federal funding to Duchenne began to include the Congressionally Directed Medical…

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Added by Annie Kennedy on June 16, 2016 at 11:00am — No Comments

National 21st Century Cures Act/ Senate Cures Day of Advocacy - TODAY!

As you know, PPMD has been helping to lead national advocacy efforts around the 21st Century Cures/ Senate Innovation legislation for more than two years and we now have a very short window of opportunity in which the…

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Added by Annie Kennedy on June 7, 2016 at 1:00pm — No Comments

Update from April 2016 Muscular Dystrophy Coordinating Committee (MDCC) Meeting

It's been a rough few weeks for this community, but our efforts to shape and reshape access barrier issues for our community continue -- and last Wednesday we took a giant leap forward. It was a pivotal day that I was proud to be a part of!



The federal intra-agency…

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Added by Annie Kennedy on May 3, 2016 at 10:28am — 1 Comment

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