Annie Kennedy's Blog (85)

Duchenne History in the Making — and We Need You to Join Us! | January 22, 2016 | Silver Spring, MD

Next week, on January 22nd, an FDA Advisory Committee will review eteplirsen for potential approval and use within our Duchenne community.

The Duchenne community is joining forces in a show of strength to demonstrate to the FDA our community’s support for our robust therapeutic pipeline. All Duchenne community members are encouraged to attend the Advisory…

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Added by Annie Kennedy on January 13, 2016 at 11:30am — No Comments

The Big Day is Almost Here!

For those of us who’ve been in this community for decades, at some point we began to believe that we’d never see the day when products that had been designed specifically with the intent to treat individuals with Duchenne would be reviewed by the FDA. But here we are—standing on the shoulders of the…

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Added by Annie Kennedy on November 18, 2015 at 11:00am — No Comments

Help Us Protect Your Wheelchair Technology Access – Act Now to Close a CMS Loophole!

Many of you have seen – and signed – a petition that circulated recently about preventing Medicare from applying competitive bidding prices to complex technology; an action that will likely reduce access to wheelchair features many of our family members depend on. This effort has now evolved into a legislative effort and we urge all members of our…

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Added by Annie Kennedy on November 3, 2015 at 10:00am — No Comments

PPMD Urges Senate to Move Forward on #Cures2015 Innovation for Healthier Americans Bill

Yesterday, I had the distinct privilege of representing PPMD and our Duchenne community as I sat before a full room of Senate staffers and congressional aides in the Dirksen Senate office building. Our goal was to urge them to press forward on the Innovation for Healthier Americans bill (also referred to as the ‘Innovation…

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Added by Annie Kennedy on October 9, 2015 at 12:00pm — 1 Comment

Industry partners receive FDA’s Pediatric Rare Disease Designation – and YOU helped make it possible!

In the last few weeks we’ve heard the news from several companies working to develop therapies for Duchenne that they have received Rare Pediatric Disease Designations from the FDA. Santhera, Sarepta, Catabasis, and Biomarin.

 

We as a community have celebrated this news. But what does it actually mean?

 

And did you know that it…

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Added by Annie Kennedy on October 7, 2015 at 4:30pm — No Comments

Meet Our Newest PPMD Adult Advisory Committee Member: Jen Petrusko Bauer, manifesting carrier

There are moments in life that take your breath away. When you are truly in awe of the power and grace of others.

 

About a year ago, we put our a call to our community asking for adults living with Duchenne and Becker to consider applying to be a part of our Innagural PPMD Adult Advisory Committee (PAAC). We were overwhelmed by the response we received and were thrilled by the…

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Added by Annie Kennedy on September 16, 2015 at 11:23am — No Comments

Join the 2016 PPMD Adult Advisory Committee – Apply by October 23!

We’re Looking for a Few (More) Good Men!

A year ago, we had a vision that we would begin to formalize the adult voice of our Duchenne community by convening a leadership group comprised of young adults and adults of accomplished and passionate men who also had Duchenne and…

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Added by Annie Kennedy on September 16, 2015 at 11:00am — No Comments

Webinar Recording: How FDA Ad Comms Work & What Role You Can Play

Ad Comms – What’s the buzz all about? If you’ve been anywhere near social media lately, you’ve likely seen the term ‘Ad Comm’ referenced. Speculation about dates, implications for approvals and access to therapies we all care about, questions about…

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Added by Annie Kennedy on September 14, 2015 at 12:32pm — No Comments

Participate in ABLE National Resource Center Survey

Since the enactment of the Stephen Beck, Jr., Achieving a Better Life Experience Act of 2014 (ABLE Act) in December 2014, PPMD has been working with leading disability organizations to ensure that states adopt and effectively…

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Added by Annie Kennedy on July 20, 2015 at 12:00pm — No Comments

PPMD's 2015 Duchenne/Becker Transitions Meeting Recap

“We are the population that was hoped for — and now we’re here.”

- L. Vance Taylor



This statement made by a close friend of mine perfectly captures the spirit that satiated the room at the PPMD Duchenne/Becker Transitions Meeting which took place at this year's Connect Conference. For two days, teens, young adults, parents,…

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Added by Annie Kennedy on July 13, 2015 at 11:00am — 1 Comment

21st Century Cures Passes House of Representatives!

21 Century Cures. First a bold concept led by two visionary congressionary leaders on April 30th, 2014.

Then innovative ideas poured in to Washington from the world’s best and brightest developers and innovators.

Then lots of Hearings, and roundtables, and Hill meetings, and constituent visits.

Then ‘provisions’ were structured which pulled…

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Added by Annie Kennedy on July 10, 2015 at 11:35am — 2 Comments

Update on 21st Century Cures Initiative



Over the last year, we have been telling you about the 21st Century Cures Initiative, a comprehensive look launched by Congress to explore what could be done to accelerate the pace of cures in American therapeutic development.…

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Added by Annie Kennedy on June 15, 2015 at 2:07pm — No Comments

Accessing Drugs Once They’re Approved

As the approval of therapies in Duchenne becomes more of a reality for our community, access becomes critical. Potential challenges with insurance coverage and approvals, as well as specialty pharmacy access begin to become a very real concern. And typically new and innovative therapies marketed to small patient communities are very expensive. Lingering…

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Added by Annie Kennedy on June 15, 2015 at 1:30pm — No Comments

Update from the PAAC: “We’re all hooked into one world now!”

On January 15 of this year we introduced you to an incredibly special group of people, PPMD’s Adult Advisory Committee. The PPMD Adult Advisory Council (PAAC) serves to ensure that the voices of adults living with Duchenne and Becker muscular dystrophy…

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Added by Annie Kennedy on March 16, 2015 at 11:00pm — No Comments

Spring Forward: Bringing the MD-CARE Act to Life

Despite great odds in a political climate that was stacked against us – we achieved what was considered to have been impossible. After months of outreach to congressional champions and visits to Capitol Hill, the MD-CARE Act Amendments were passed and signed into…

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Added by Annie Kennedy on March 15, 2015 at 12:04pm — No Comments

We Need YOU to Help Us With the Development of the Solid Suit

In December, we announced the launch of an exciting collaboration between PPMD, Solid Ventures, and SRI International to develop the ‘Solid Suit’. The Solid Suit is…

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Added by Annie Kennedy on March 2, 2015 at 2:30pm — 1 Comment

Meet our 2015 PPMD Adult Advisory Council!

“We were the generation that was hoped for… And now we’re here.” L. Vance Taylor

The PPMD Adult Advisory Council (PAAC) serves to ensure that the voices of adults living with Duchenne and Becker muscular dystrophy are reflected in…

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Added by Annie Kennedy on January 15, 2015 at 1:00pm — No Comments

The Solid Suit: Combining Forces to Enhance Strength

(Pictured from left to right: Stanley Nelson, MD, Center for Duchenne Muscular Dystrophy at UCLA, Thomas Sugar, PhD, Arizona State University Polytechnic School, Tom Egan, SRI International, Roy Kornbluh, SRI International, Laura Case, DPT,…

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Added by Annie Kennedy on December 18, 2014 at 9:57am — 1 Comment

PPMD Holds Congressional Briefing

It was seven months to the day that Representatives Upton and DeGette first launched this bold initiative called 21st Century Cures. In fact, that hearing that would set in motion months and months of proposals for sweeping changes to research infrastructure and academic systems took place just across the hall from where we were now gathered. PPMD,…

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Added by Annie Kennedy on December 16, 2014 at 9:30am — No Comments

ABLE Act Changes Disability Landscape – This just got real!

If you’re like many families, birthdays and holidays often bring gifts from friends and loved ones far and wide. As my son just celebrated his fourth birthday, he would tear through packages in the hope of finding a new Lego set or Ninja Turtle. He was a bit less interested in the envelopes that accompanied the gifts but patiently waited as Mommy read the cards to him and announced who each gift was from. And the concept of monetary gifts was a bit lost on him. The…

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Added by Annie Kennedy on December 3, 2014 at 6:00pm — No Comments

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