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Staff
PPMD’s #EverySingleOne Tour: Gainesville, FL

PPMD's Pat Furlong 

 

Earlier this month, we had the privilege of having our fourth stop in 2017 on …

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Added by PPMD on June 14, 2017 at 10:30am — No Comments


Staff
PTC Therapeutics Announces FDA Advisory Committee Meeting for Ataluren

PTC Therapeutics, Inc. today announced that the FDA has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna™). This is another important moment for our community and we look forward to the opportunity to share our collective experiences with Translarna with the FDA. As details on this Ad Comm become…

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Added by PPMD on June 6, 2017 at 8:47am — No Comments


Staff
DuchenneConnect Expands Down Under!



Last year advocates from Save Our Sons Duchenne Foundation (SOS) approached PPMD about partnering to have a patient report registry in Australia. They were worried because at the time there were no…

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Added by Ann Lucas on June 5, 2017 at 9:30am — No Comments


Staff
PTC Therapeutics Provides EMFLAZA™ (Deflazacort) FAQ Update

PTC Therapeutics has provided an update regarding EMFLAZA (see below). Please visit EMFLAZA.com for answers to some of the most frequently asked questions PTC has…

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Added by PPMD on June 2, 2017 at 11:30am — No Comments


Staff
Get in the Game with Coach To Cure MD on September 30th!

Over the last 9 years, Coach To Cure MD has raised $1.5 million thanks to families like yours! Every dollar you have raised has helped us advance promising research, which led to two drug approvals for Duchenne in just the last year…

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Added by Danielle Garrigan on June 1, 2017 at 3:30pm — No Comments


Staff
FDA Makes Determination Regarding In-Dwelling Ports

Time and again in our Duchenne community, we see brave kids and their warrior parents work to change the landscape. After all, it’s that exact spirit upon which PPMD was founded. And with that same spirit that family after family has transformed their personal moments into broader movements that have benefited us all.

 

On May 18, the FDA Pediatric Advisory Committee convened to determine whether to allow for the protocol of Sarepta’s ESSENCE trial to…

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Added by Annie Kennedy on May 26, 2017 at 2:45pm — No Comments


Staff
Finding Your Light: Fighting Depression in Duchenne

PPMD's Kathi Kinnett, MSN, CNP worked with Molly Colvin, PhD, Neuropsychologist at Massachusetts General Hospital and Ginny Ward, an amazing mom living in Colorado with her children, including John, a young man living with Duchenne, to develop this important material. …

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Added by Kathi Kinnett on May 25, 2017 at 11:00am — No Comments


Staff
Before Summer Kicks Off, Get Your Event Kicked Off!

The Race to End Duchenne .1K is the perfect school event to celebrate Duchenne Action Month! About the length of a football field, a .1K is the race anyone can do! It requires no training and is…

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Added by Nicole Herring on May 24, 2017 at 12:52pm — No Comments


Staff
FDA Pediatric Advisory Committee Unanimously Votes YES — Now Moves to Commissioner

Today’s Pediatric Advisory Committee Meeting resulted in a unanimous decision from committee members to recommend the use of in-dwelling ports in Sarepta's ESSENCE…

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Added by Annie Kennedy on May 18, 2017 at 4:30pm — No Comments


Staff
Today’s FDA Pediatric Advisory Committee Meeting

As our clinical trial pipeline unfolds and therapies become available, navigating the landscape is becoming increasingly complex. But today our landscape shone a bit brighter as the sun rose on the FDA campus early this morning and we watched members of our Duchenne community…

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Added by Annie Kennedy on May 18, 2017 at 10:00am — No Comments


Staff
PPMD Publishes & Testifies on Newborn Screening

PPMD's SVP of Legislation & Public Policy, Annie Kennedy and Michelle Puryear, MD, PhD, testified at the Advisory Committee on Heritable Disorders in Newborns and Children last week.



On the heels of a recent publication in the International Journal of Neonatal Screening, Dr. Puryear provided an update of the therapeutic pipeline and some of PPMD's activities around developing infrastructure to support a pilot for newborn screening for…

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Added by Annie Kennedy on May 16, 2017 at 4:40pm — No Comments


Staff
PPMD Signs on to FDA User Fee Letter

PPMD is proud to join dozens of other rare disease nonprofit organizations in asking Congressional leaders to reauthorize the FDA user fee agreements.

The current FDA user fee agreements are the culmination of months of negotiation between FDA and the medical product industry, with significant input from the patient advocacy community. 

Read the…

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Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments


Staff
PPMD Helps Author Expanded Access Principles

Earlier this month, PPMD was proud to be among seven patient advocacy organizations who took a lead in laying out a joint set of principles to guide any efforts that seek to change the process of accessing unapproved therapies outside of a clinical trial, also known as compassionate use or expanded access.

The patient advocacy organizations include:

  • Alliance for Aging Research
  • American Cancer Society Cancer Action Network
  • Friends of…
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Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments


Staff
Senate Introduces BENEFIT Act

PPMD is thrilled to announce that on May 4 the BENEFIT Act was introduced in the Senate by longtime Duchenne community champions, Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN).

 

Over the past five years, Congress has made considerable progress in driving forward policies and procedures to ensure the patient perspective is considered by Food and Drug Administration (FDA) reviewers evaluating candidate drugs and other medical products. As a result of…

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Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments


Staff
2017 DRSC Annual Meeting

On May 9, the Duchenne Regulatory Science Consortium (DRSC) held its Annual Meeting in Crystal City, VA. In the audience of about 40 people, there were industry and academic Consortium members, potential industry members, five FDA representatives, two NIH representatives, C-PATH representatives, and Buddy Cassidy, the DRSC…

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Added by Abby Bronson on May 16, 2017 at 3:00pm — No Comments


Staff
Deciphering What We Know About EMFLAZA

The unknowns. Some of the hardest beasts to conquer in life.

 

The unpredictable variables that make life impossible to plan.

 

The not being able to commit to vacations and holiday plans because – well, you just don’t know what curve balls life will toss at you between…

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Added by Pat Furlong on May 15, 2017 at 9:30am — 1 Comment


Staff
In Honor of Moms: A Note from Tom Furlong

This Mother’s Day, thank someone for their role in the fight to end Duchenne.

If you’re reading this blog, chances are you know my wife, Pat Furlong. For 23 years now, Pat has been leading Parent Project Muscular Dystrophy (PPMD) and the fight to end Duchenne. Many of you have told me over the years that Pat has been a source of comfort,…

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Added by PPMD on May 10, 2017 at 10:30am — No Comments


Staff
PPMD's 2017 Connect Conference Agenda is Now Available!

PPMD is excited to unveil the agenda for our 2017 Connect Conference in Chicago, June 29 - July 2, 2017!

 

We are so proud of the …

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Added by PPMD on May 9, 2017 at 11:00am — No Comments


Staff
PPMD's Response to PTC Announcement of EMFLAZA (deflazacort) Pricing & Access

Today, PTC Therapeutics provided a community update on EMFLAZA (deflazacort) which they acquired earlier in the year from Marathon Pharmaceuticals. The community has anxiously awaited an update on both access and pricing ever since the deal between PTC and Marathon was announced.



On today’s call, we…

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Added by PPMD on May 8, 2017 at 5:38pm — No Comments


Staff
PTC Therapeutics to Discuss Launch Plans for Emflaza™ (Deflazacort) during Community Call on Monday, May 8th at 3:00 PM EDT

PTC Therapeutics has released a letter to the community and will host a conference call for the Duchenne community to discuss the launch plans for EMFLAZA™ (deflazacort) on Monday, May 8, 2017 at 3:00 PM EDT (call-in details are below).



On a…

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Added by PPMD on May 8, 2017 at 10:00am — 1 Comment

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