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:
Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments
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…Continue
Added by Annie Kennedy on May 16, 2017 at 4:00pm — No Comments
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…Continue
Added by Abby Bronson on May 16, 2017 at 3:00pm — No Comments
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…Continue
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,…Continue
Added by PPMD on May 10, 2017 at 10:30am — No Comments
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 …Continue
Added by PPMD on May 9, 2017 at 11:00am — No Comments
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…Continue
Added by PPMD on May 8, 2017 at 5:38pm — No Comments
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).
The newest version of the GOP HealthCare Bill is coming to a vote by 1:30 PM EDT this afternoon!Continue
Added by Ryan Fischer on May 4, 2017 at 12:00pm — No Comments
The current version of American Health Care Act (AHCA) directly threatens essential health benefits (EHBs), Medicaid eligibility and funding, and vital health care protections for our Duchenne community.
I wish there was a way to…
Added by Annie Kennedy on April 28, 2017 at 9:30am — No Comments
PTC Therapeutics, Inc. will host a conference call for the Duchenne community to discuss the launch plans for Emflaza™ (deflazacort) on Monday, May 8, 2017 at…Continue
Added by PPMD on April 26, 2017 at 10:00am — No Comments
Capricor Therapeutics today announced positive top-line results from a safety and exploratory efficacy analysis of six-month data from the randomized 12-month Phase I/II HOPE Clinical Trial of CAP-1002 (allogeneic cardiosphere-derived cells), an investigational candidate for the treatment of patients with Duchenne.
We are excited to see these results and to see the field of cell…Continue
On May 18, 2017, FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s…Continue
Added by PPMD on April 24, 2017 at 12:30pm — No Comments
Today, PTC Therapeutics announced that they have completed their acquisition of Emflaza™ (deflazacort) ahead of schedule, following early conclusion of the anti-trust review.
PTC will be hosting a call with the Duchenne community the week of May 8 to provide details on the plan for the launch of Emflaza. We look forward to learning more from PTC in the…Continue
Added by PPMD on April 20, 2017 at 4:00pm — No Comments
The Duchenne community has waited 20 years to be where we are. We are reminded daily of how far we have come, how the landscape has changed, how there are close to 20 clinical trials to choose from. While these changes within are landscape bring us to a hopeful time in Duchenne, the journey to…Continue
Added by Kathi Kinnett on April 20, 2017 at 12:30pm — No Comments
Great news – The ABLE program enhancements have been re-introduced in Congress and are gaining support. This package of bills include improvements to the original ABLE program that directly impact our community, here’s how:
On April 4th, a bi-partisan group of Members of Congress,…Continue
Added by Annie Kennedy on April 14, 2017 at 9:00pm — No Comments
In late March, PPMD joined 87 organizations to issue a letter calling on Congress to take a hard look at the likely significant and…Continue
Added by Annie Kennedy on April 13, 2017 at 9:30pm — No Comments
PPMD is proud to sign on to letters sent both to the Senate and the House asking that they provide at least $29.8 million to the Centers for Disease Control and Prevention’s (CDC) Newborn Screening Quality Assurance Program (NSQAP) and at least $19.9 million to the Health Resources and Services…Continue
Added by Annie Kennedy on April 12, 2017 at 9:00pm — No Comments
Iowa Children’s Hospital marked our third stop on PPMD’s 2017 Every Single One Tour, held on April 1, 2017. The amazing staff at Iowa Children’s Hospital helped make this a wonderful day and we can see…Continue
Added by PPMD on April 7, 2017 at 1:30pm — No Comments
Today Parent Project Muscular Dystrophy (PPMD) named UC Davis Department of Physical Medicine and Rehabilitation Neuromuscular Disease Clinic (UC…Continue
Added by PPMD on April 6, 2017 at 12:30pm — No Comments