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
Viral gastrointestinal (GI) viruses are no fun for anyone, but they are especially worrisome for a person living with Duchenne muscular dystrophy. GI viruses affect the GI track – the stomach and intestine (i.e., ‘gut’) – resulting in abdominal pain/discomfort, nausea, vomiting, intestinal pain/discomfort, cramps and diarrhea. Thank goodness they usually have…Continue
Added by Kathi Kinnett on December 21, 2017 at 10:30am — No Comments
For the last many months, we have all been watching gene therapy make headlines around the world, across various diseases. As you know, earlier this year PPMD launched a Gene Therapy Initiative to help support the exploration of various gene therapy technologies in Duchenne.
Of particular note has been the progress made in…Continue
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
We’ve entered a new chapter for families with Duchenne, and 2017 has been a year of incredible promise.
Yes, we’ve faced some setbacks, but we’ve also seen incredible successes, like recent breakthroughs in gene therapy. It’s essential that we keep…
Added by Abby Bronson on December 12, 2017 at 9:00am — 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
This holiday season, PPMD is asking you to support our ongoing Gene Therapy Initiative. Gene therapy has dominated the headlines this year in a number of diseases, including Duchenne. There is such…Continue
Added by Pat Furlong on December 7, 2017 at 10:30am — 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
An email from a mom in war-torn Baghdad who doesn’t know how to even start to seek care for her son with a new diagnosis.
A mom in India who can’t find a provider who will give her son steroids, but instead say stem cells will cure him.
A physical therapy student in Turkey who wants to learn all she can…Continue
Added by Kathi Kinnett on December 5, 2017 at 11:55am — No Comments
The Senate passed their tax reform bill. In the dead of night. Overnight -- shortly before 2 AM on Saturday.
Despite protests and outcries from almost every sector: non-profits, disability communities, healthcare providers, educators, universities, researchers, graduate students (future researchers, geneticists, nurses,…Continue
Added by Annie Kennedy on December 5, 2017 at 9:00am — No Comments
So much has changed in the Duchenne research landscape since we were founded back in 1994, but your commitment has been a constant since our story began.
You’ve never stopped fighting, and the promise for our community has never been greater, with recent breakthroughs in gene therapy, one of the most exciting approaches we’ve ever seen. Today, we…
Added by Pat Furlong on December 4, 2017 at 10:30am — No Comments
Throughout the history of Duchenne, there have been moments that we recognize as inflection points, times when we saw a significant shift in the fight to end Duchenne, when the…Continue
Added by Abby Bronson on November 30, 2017 at 6:30pm — 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