I am a 22 year old living with Duchenne, and opportunities for clinical trial participation are few and far between. It is one of the frustrating realities of my diagnosis and my age. Although it has never swayed my commitment to this community, I have to be honest. It can be discouraging at…
Added by PPMD on December 30, 2017 at 10:03am — No Comments
I have some exciting news: Your response to Sean and Mindy Rice’s generous matching gift of $200,000 has been incredibly inspiring to the Parent Project Muscular Dystrophy board. You’ve shown us that PPMD’s Gene Therapy Initiative matters to you, and we want to keep up the momentum.…
Added by PPMD on December 26, 2017 at 3:00pm — No Comments
My son Bazi is four years old and has Duchenne muscular dystrophy. It's been over a year now, and this sentence is still extraordinarily hard for me to write.
When we got the diagnosis, my emotions shifted between paralyzing fear, incredible…Continue
Added by PPMD on December 22, 2017 at 11:30am — No Comments
When our son, Jonathan, was diagnosed with Duchenne, we didn’t know where our family would find the strength to fight. But the PPMD community has always shown us that we are never alone in our fight to end Duchenne.
Added by PPMD on December 19, 2017 at 2:56pm — No Comments
With advancements made in Duchenne gene therapies now coming to fruition, a number of questions are being asked about who may qualify for the upcoming trials.
While the inclusion criteria of each trial will be different based on age, mutation (in some cases), and functional abilities, one of the criteria for participating in…Continue
PTC Therapeutics provided a brief update on the Formal Dispute Resolution Request process for ataluren.
Read the update from PTC:
As we enter the holiday season, I wanted to thank the community for all your support throughout the regulatory…
Added by PPMD on December 13, 2017 at 11:00am — No Comments
Santhera has provided an update to the Duchenne community, which contains updates on the SIDEROS study, educational events, respiratory guidelines/care, and more. Click here to view the update.…
Added by PPMD on December 11, 2017 at 4:30pm — No Comments
PPMD joined 153 members of the CDC Coalition and other supporting state and national organizations in urging Congressional leaders to provide at least $7.8 billion for the Centers for Disease Control and Prevention’s programs as part of the final FY 2018 Labor, Health and Human Services, Education and Related Agencies Appropriations bill and other final FY…Continue
Added by PPMD on December 7, 2017 at 12:20pm — No Comments
Parent Project Muscular Dystrophy, FSH Society Lead Effort to Obtain
Critical Diagnostic Classification Standard
Parent Project Muscular Dystrophy (PPMD) along with collaborators the…Continue
Added by PPMD on December 7, 2017 at 8:30am — No Comments
Today Parent Project Muscular Dystrophy (PPMD) named UCSF Benioff Children’s Hospital San Francisco (UCSF) our 18th Certified Duchenne Care Center.
PPMD’s Certified Duchenne Care Center Program, which…Continue
Added by PPMD on December 6, 2017 at 9:30am — No Comments