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All Blog Posts Tagged 'Staff' (750)


Staff
PPMD Convenes International Experts for Inflammation and Immunity in Duchenne Muscular Dystrophy Workshop

Today, PPMD is convening medical experts from around the world for a workshop exploring inflammation and immunity in Duchenne on the eve of our 23rd Annual Connect Conference.



In January, PPMD organized a Critical…

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Added by PPMD on June 28, 2017 at 2:19pm — No Comments


Staff
PPMD Research Update: June 2017

An Overview of Your Impact in Action



We are less than a week away from PPMD’s Annual Connect Conference, where researchers and industry from around the world will provide…

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Added by Abby Bronson on June 23, 2017 at 2:00pm — No Comments


Staff
PPMD Launches New Resource Center for Families

Navigating life with Duchenne can be daunting and time consuming for families. We rely on each other to share wisdom and information in order to make the process smoother and life a bit easier. The best guidance we can get is from those who have been through a given situation, sharing what they’ve learned to help others navigate resources that exist.

 

PPMD strives to find…

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Added by Ryan Fischer on June 21, 2017 at 10:00am — No Comments


Staff
FDA Hiring Freeze Lifted

Recently, FDA Commissioner Scott Gottlieb announced that the hiring freeze, instituted on January 23, has been lifted.

This reversal means that the FDA can begin to fill vacancies – needed resources for continued research into rare disease treatments and for the review and…

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Added by PPMD on June 15, 2017 at 11:53am — No Comments


Staff
65 Members of Congress Send Letter of Strong Support for Duchenne Priorities for Next Year’s Budget

With Congress not wrapping up spending bills for Fiscal Year 2017 until we were more than 7 months into the fiscal year, the FY 2018 budget and appropriations (funding) cycle will be interesting to say the least. The Trump Administration released a preliminary — or “skinny” budget -- in March but…

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Added by Ryan Fischer on June 15, 2017 at 11:30am — No Comments


Staff
PPMD Signs On to DoD Funding Letters

Once again, PPMD has signed on to letters to leaders in both the House and Senate, requesting they continue to support the critical and highly successful defense health research programs funded through the Congressionally Directed Medical Research Programs (CDMRP) at the…

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Added by PPMD on June 15, 2017 at 11:30am — No Comments


Staff
Celebrate Dad & Your Gift is Doubled!

This Father’s Day, I hope you will join me in celebrating the dads in this community. They are often the unsung heroes of our community. They are role models for our sons, providing for our families. Their hearts ache just as profoundly when our children are hurting. Their hearts love just as unconditionally.

That’s why this Father’s Day, I…

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Added by Pat Furlong on June 14, 2017 at 12:30pm — No Comments


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
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
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

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