PPMD's Blog (474)

Two days left to reach our goal

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

Great News: Match extended by PPMD board!

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

Gene therapy gives me the courage to hope

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…


Added by PPMD on December 22, 2017 at 11:30am — No Comments

We're doubling your donation for gene therapy


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

Understanding Gene Therapy: The Screening Process

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…


Added by PPMD on December 13, 2017 at 4:00pm — 1 Comment

PTC Therapeutics Shares Update on Ataluren Formal Dispute Resolution Request Process

PTC Therapeutics provided a brief update on the Formal Dispute Resolution Request process for ataluren.

Read the update from PTC:

Dear Duchenne Community,

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 Update for Duchenne Community on SIDEROS Study

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 Signs Letter to Save CDC

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…


Added by PPMD on December 7, 2017 at 12:20pm — No Comments

Duchenne/Becker and FSH Muscular Dystrophies Receive ICD-10 Codes

Parent Project Muscular Dystrophy, FSH Society Lead Effort to Obtain

Critical Diagnostic Classification Standard


Parent Project Muscular Dystrophy (PPMD)  along with collaborators the…


Added by PPMD on December 7, 2017 at 8:30am — No Comments

PPMD Designates UCSF Benioff Children's Hospital San Francisco a Certified Duchenne Care Center

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…


Added by PPMD on December 6, 2017 at 9:30am — No Comments

Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Duchenne

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…


Added by PPMD on November 30, 2017 at 10:24am — No Comments

Capricor Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application for CAP-1002

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…


Added by PPMD on November 29, 2017 at 8:30pm — No Comments

Capricor Reports Significant Improvements in Duchenne Patients Treated with CAP-1002 Investigational Cell Therapy

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…


Added by PPMD on November 15, 2017 at 2:30pm — 1 Comment

PPMD Submits Community Sign-On Letter to FDA Supporting Approval of Ataluren

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 Initiates Clinical Trial for Lead Program in Duchenne

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…


Added by PPMD on November 6, 2017 at 9:09am — No Comments

Sarepta & Nationwide Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne

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…


Added by PPMD on November 6, 2017 at 8:30am — No Comments

Sarepta Therapeutics & Nationwide Children’s Hospital Announce FDA Clearance of the IND Application for the GALGT2 Gene Therapy Program

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…


Added by PPMD on November 3, 2017 at 8:39am — No Comments

Letter from PTC Therapeutics to the Community Following FDA Decision

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…


Added by PPMD on October 26, 2017 at 9:30am — No Comments

News from 2017 World Muscle Society: Part 2

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…


Added by PPMD on October 9, 2017 at 12:30pm — No Comments

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