Featured Blog Posts (384)


Staff
Just One Month Away from the 9th Annual Coach to Cure MD!

Join us September 24th as thousands of coaches from hundreds of universities wear the Coach To Cure MD patch on their sleeve to raise awareness. Families will join teams on the field on game day and will share their stories with spectators and media. We also…

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Added by PPMD on August 24, 2016 at 12:30am — No Comments


Staff
PPMD Sends Letter to FDA Regarding Duchenne Therapies



Today, PPMD sent a letter to the FDA requesting updates on various regulatory actions of tremendous importance to the Duchenne community. In our letter, we are asking the FDA to talk…

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Added by PPMD on August 11, 2016 at 12:00pm — No Comments


Staff
Every Single Family

In the 20+ years PPMD has been around, the average lifespan of people with Duchenne has increased. This incredible leap is not because of any drug interventions or treatments because as we all know, to date there are no approved therapies. Rather this is because of the push from PPMD to advance care over the last two decades.

PPMD strives to ensure that every single person with Duchenne receives optimal care. …

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Added by PPMD on July 28, 2016 at 2:18pm — No Comments


Staff
Thank you to our 2016 Connect Conference Resource Fair Providers!

The 2016 Connect Conference again provided a forum for families to interact with a variety of resource providers. We thank our resource fair sponsors for working with us to make every single resource known to our community!…

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Added by PPMD on July 14, 2016 at 1:36pm — No Comments


Staff
Getting Results: Success from your Advocacy efforts in February!

Thanks to your advocacy, this week we learned that all of the Duchenne priorities we advocated for this year with Members of Congress are one step closer to being included in next year’s budget.

 

Whether you traveled to DC for face-to-face meetings with members, or advocated from…

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Added by Ryan Fischer on June 10, 2016 at 12:00pm — No Comments


Staff
Sarepta Announces FDA Will Not Complete the Review of the Eteplirsen NDA By The PDUFA Date

The FDA has notified Sarepta that they are continuing their review of eteplirsen and will not be able to complete…

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Added by PPMD on May 25, 2016 at 12:00pm — No Comments


Staff
PPMD Names Children’s Mercy Hospital in Kansas City, MO, Certified Duchenne Care Center

PPMD Awards Eleventh Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care

 

Children’s Mercy Hospital (Children’s…

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Added by PPMD on May 16, 2016 at 2:08pm — No Comments


Staff
PPMD's 2016 Connect Conference Agenda Now Available!

PPMD is excited to unveil the agenda for the 22nd Annual Connect Conference in Orlando, June…

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Added by PPMD on April 11, 2016 at 1:16pm — No Comments


Staff
How You Can Help with the Upcoming Ad Comm for Eteplirsen – Every Single Voice Matters!

We are just a few short weeks from the FDA Advisory Committee Meeting for eteplirsen on April 25th. PPMD is part of a united coalition of Duchenne organizations (MakeDuchenneHistory Coalition) working together to ensure we make the…

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Added by Ryan Fischer on April 4, 2016 at 10:30am — 1 Comment


Staff
PPMD is Initial Partner in OnPAR — Online Partnership to Accelerate Research

The National Institutes of Health (NIH) is one of the biggest funders of medical research in the US, and projects it has funded have touched almost every Duchenne therapeutic in development now in some way. In FY2016, the NIH…

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Added by Abby Bronson on March 24, 2016 at 9:30am — No Comments


Staff
FDA Releases New Date for Eteplirsen Ad Comm

At long last we have received information regarding the rescheduled Advisory Committee for Sarepta (eteplirsen). The Ad Comm will take place on Monday, April 25 at the College…

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Added by PPMD on March 10, 2016 at 9:00am — No Comments


Staff
Gene Therapy Today: Notes from PPMD’s Duchenne Gene Therapy Forum



Gene therapy.

Those two words have been tossed around for some time, starting in the late 1990s when several gene therapy trials started, but ended with fatal results. Now, when the field has learned a lot from those initial results and with optimized technologies, we come back to gene…

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Added by Abby Bronson on March 8, 2016 at 12:00pm — 4 Comments


Staff
PPMD Launches the Every Single [One] Tour

For 22 years, PPMD has brought together families with leading experts, care providers, and industry partners through our Annual Connect Conference,…

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Added by PPMD on March 4, 2016 at 10:00am — 1 Comment


Staff
Congress Calls on FDA to Increase Efforts to Treat Duchenne



Thanks to this incredible community urging lawmakers, well over 100 Members of Congress signed and submitted a letter …

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Added by Ryan Fischer on February 18, 2016 at 2:40pm — 1 Comment


Staff
PPMD Announces Gene Therapy Forum

This year’s already action-packed Advocacy Conference, February 21-23 welcomes a special Gene Therapy Forum on Monday, February…

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Added by Abby Bronson on February 10, 2016 at 10:30am — 2 Comments


Staff
Advancing Targeted Therapies for Rare Diseases Act Goes Before HELP Committee

For nearly two years, PPMD has been working with Congressional champions and industry partners to help lead an effort to streamline the regulatory pathways and review processes for targeted therapeutics (which would include such things as ‘follow on exons’) by clarifying the FDA’s existing authority to leverage data previously used in the approval of a targeted product when approving a new therapy. These efforts have yielded a provision in the House of Representatives…

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Added by Annie Kennedy on February 9, 2016 at 3:36pm — 8 Comments


Staff
Growing Our Team

We are not a team because we work together. We are a team because we respect, trust, and care for each other. – Vala Afshar

 

I am the first person to admit that I don’t know much about a lot of different things. But I do know talent when I see it. And I could not be…

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Added by Pat Furlong on February 1, 2016 at 9:43am — No Comments


Staff
ACTION ALERT: Sign-On Extended! Urge House Member - Apply FDASIA Tools to Duchenne

Action Alert

PPMD and a coalition of Duchenne foundations continue to work together on preparing for the Sarepta Ad Comm. As of today, we are still waiting on a new confirmed date. With the postponement of the Ad Comm, the Congressional letter to FDA remains open. Has your House Members signed on…

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Added by Ryan Fischer on January 25, 2016 at 11:30am — No Comments


Staff
ACTION ALERT: Urge House Member - Apply FDASIA Tools to Duchenne

There has never been a moment like this in our Duchenne community. Today the FDA released briefing documents on the application for eteplirsen. It is clear that we continue to face a serious regulatory challenge as a community.…

Action Alert

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Added by Ryan Fischer on January 15, 2016 at 2:45pm — 1 Comment


Staff
We did it! Over $270,000 raised to support early-stage research.

Last month we told you we hoped to raise $200,000 to support new, early-stage research. Throughout the month, we told you about potential therapies like epicatechin. We told you about generous matches from Mary-Lou and Larry Weisman and the Dumm Family/…

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Added by Pat Furlong on January 5, 2016 at 11:00am — No Comments

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