Yesterday, Kymriah, a gene therapy made by Novartis that treats an aggressive type of leukemia was approved for children and young adults. The new commissioner of the FDA, Dr. Scott Gottlieb,…Continue
Added by Abby Bronson on August 31, 2017 at 1:00pm — No Comments
Added by Kathi Kinnett on August 28, 2017 at 11:30am — No Comments
With trials in gene therapy on the horizon, and terms like "cassette", "construct", "promoter", and "transduce" being thrown around, we want to ensure that you, our Duchenne Community, has all the knowledge you need to navigate the world of gene therapy.
Added by Abby Bronson on August 23, 2017 at 8:00pm — No Comments
The Duchenne community is now preparing for the September 28 Advisory Committee Meeting for PTC’s therapy ataluren. Whether submitting written…Continue
Added by PPMD on August 22, 2017 at 11:00am — No Comments
May 12-13, 2016, PPMD convened a workshop of 48 opinion leaders, research experts and clinicians from the US and Canada, on the subject of Duchenne and bone biology. Bone health has long been a…Continue
Added by Kathi Kinnett on August 14, 2017 at 10:00pm — No Comments
Hope is critical to survival. Hope helps us get through those times of darkness with the thought that there is something new on the horizon, a potential new destination. Hope enables us to focus on meeting challenges even in the midst of adversity. Hope allows us to see that there might be…Continue
Added by Ann Lucas on August 7, 2017 at 2:00pm — No Comments
The PDUFA VI bill – known as FDA Reauthorization Act in the House (FDARA) – passed the House of Representatives early in July with strong bi-partisan support. It then moved to the Senate but consideration was delayed until after the Senate healthcare reform deliberations were completed. Today, though, we are excited that a companion bill (mirroring the House version) passed in the Senate! It will now move to the President’s desk where it is expected…Continue
Added by Annie Kennedy on August 3, 2017 at 8:30pm — No Comments
Added by Abby Bronson on July 31, 2017 at 10:46am — No Comments
Yesterday the Senate voted 51-50 to move forward on healthcare reform legislation -- but no specific bill was actually voted on. The Senate has committed to passing something this week. Once passed, the Senate bill will then have to return to the House for passage or conferencing (if it differs from…Continue
Added by Annie Kennedy on July 26, 2017 at 9:00am — No Comments
In April 2016, PPMD convened a workshop to assess the measures used to evaluate pulmonary strength, air flow and lung volumes. Thanks to the tireless effort of Dr. Jonathan Finder, this summary has now…Continue
Added by Kathi Kinnett on July 17, 2017 at 9:35am — No Comments
Sadly, I never had the honor of meeting PJ Nicholoff, but I’ve met his parents. And if PJ was anything like his father, Brian, he was vibrant, compassionate, caring, engaged and tenacious!
Added by Kathi Kinnett on July 16, 2017 at 8:30pm — No Comments
Today Parent Project Muscular Dystrophy (PPMD) named Gillette Children's Specialty Healthcare Neuromuscular Clinic (Gillette) our 16th …Continue
Added by PPMD on July 13, 2017 at 11:21am — No Comments
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…Continue
Added by PPMD on June 28, 2017 at 2:19pm — No Comments
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…Continue
Added by Abby Bronson on June 23, 2017 at 2:00pm — No Comments
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…Continue
Added by Ryan Fischer on June 21, 2017 at 10:00am — No Comments
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…Continue
Added by PPMD on June 15, 2017 at 11:53am — No Comments
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…Continue
Added by Ryan Fischer on June 15, 2017 at 11:30am — No Comments
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…Continue
Added by PPMD on June 15, 2017 at 11:30am — No Comments
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…Continue
Added by Pat Furlong on June 14, 2017 at 12:30pm — No Comments
PPMD's Pat Furlong
Earlier this month, we had the privilege of having our fourth stop in 2017 on …Continue
Added by PPMD on June 14, 2017 at 10:30am — No Comments