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
Solid Biosciences announced today that it has initiated clinical trial activities for SGT-001, the company’s lead microdystrophin gene transfer candidate for the treatment of Duchenne.
The Phase I/II adaptive study, called IGNITE DMD, will evaluate the safety and efficacy of a single intravenous (IV) dose of SGT-001 in ambulatory and non-ambulatory adolescents and children with Duchenne. Enrollment will begin at the first clinical trial site in the United…Continue
Added by PPMD on November 30, 2017 at 10:24am — No Comments
Capricor Therapeutics today announced that the FDA has cleared its Investigational New Drug (IND) application to conduct a new clinical trial of CAP-1002, its lead investigational therapy, in boys and young men in advanced stages of Duchenne. PPMD looks forward to being join by Capricor for a community webinar tomorrow, November 30 at 1:00 PM EST…Continue
Added by PPMD on November 29, 2017 at 8:30pm — No Comments
PPMD is excited to learn that Capricor's investigational cell therapy, CAP-1002, is showing significant improvements in people with Duchenne. We look forward to learning more details about this therapy in a community webinar on Wednesday, November 29 at 1pm eastern. Details coming soon!
Read the release from…Continue
PPMD has been continuing to engage with both PTC and the FDA on behalf of the patient community regarding the regulatory review of ataluren.
Earlier this month, PPMD reached out to FDA requesting guidance on…
Added by PPMD on November 13, 2017 at 1:00pm — No Comments
Wave Life Sciences Ltd. announced the initiation of a global Phase 1 clinical trial for WVE-210201 in Duchenne patients amenable to exon 51 skipping. PPMD is excited by the progress Wave Life Sciences has made in exon skipping. While advances have been made in exon skipping, especially recently, there is more to explore within this technology. We…Continue
Added by PPMD on November 6, 2017 at 9:09am — No Comments
More good news in gene therapy! Today, Sarepta Therapeutics announced that Nationwide Children’s Hospital has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for the micro-dystrophin gene therapy program. The Phase 1/2a clinical trial, now open to enrollment and scheduled for initiation by mid-November, is designed to assess the safety and tolerability of AAVrh74.MHCK7.micro-Dystrophin in individuals…Continue
Added by PPMD on November 6, 2017 at 8:30am — No Comments
Once again, PPMD is excited to see industry, patient groups, and research partnering, as today Nationwide Children's Hospital and Sarepta Therapeutics announced the achievement of an important milestone in…Continue
Added by PPMD on November 3, 2017 at 8:39am — No Comments
PTC has shared a letter with the Duchenne community in regards to the recent FDA decision. PTC will be holding a community call on Friday, October 27 at 12:00 PM EST to discuss next steps with the community. Please e-mail your…Continue
Added by PPMD on October 26, 2017 at 9:30am — No Comments
PPMD thanks the 40 US physicians joining the call for approval of ataluren. This letter compliments the …Continue
Added by PPMD on October 17, 2017 at 9:00am — No Comments
Annie Kennedy and Kathi Kinnett from the PPMD team recently attended the 22nd International Annual Congress of the World Muscle Society and have been reporting back the latest updates related to…Continue
Added by PPMD on October 9, 2017 at 12:30pm — No Comments