PPMD's Blog – April 2016 Archive (9)

April 25: FDA Advisory Committee Meeting for Eteplirsen - Live Webcast Information

On Monday, April 25, an FDA Advisory Committee will review eteplirsen for potential approval and use within our Duchenne community.

The Duchenne community is joining forces in a show of strength to demonstrate to the FDA our community’s support for our robust therapeutic pipeline. Over 850 Duchenne community members are scheduled to attend the…


Added by PPMD on April 21, 2016 at 8:30am — No Comments

Briefing Documents for Rescheduled Eteplirsen Ad Comm Released

The briefing documents for Sarepta’s Ad Comm on Monday, April 25th have been released. Like you, we will spend the next several days dissecting these documents so…


Added by PPMD on April 21, 2016 at 8:00am — No Comments

PPMD Advocates Ensure Congress Recognizes Importance of Flexibility in FDA Reviews

All your calls, emails, and Hill visits during our February Advocacy Conference (in the pouring rain!) are yielding GROUND BREAKING RESULTS.


Yesterday, the Federal Appropriations Report Language for…


Added by PPMD on April 19, 2016 at 4:00pm — No Comments

PPMD Names Children’s Hospital Los Angeles 10th Certified Duchenne Care Center

PPMD Awards Tenth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care

Children’s Hospital Los Angeles (CHLA) became the tenth clinic named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD). PPMD’s Certified Duchenne…


Added by PPMD on April 19, 2016 at 9:00am — No Comments

Last Day for Early Bird Registration | PPMD's 2016 Connect Conference

PPMD will host our 22nd Annual Connect Conference in OrlandoJune 26-29. This will be an incredible coming together of the community's most…


Added by PPMD on April 15, 2016 at 10:30am — No Comments

Parent Project Muscular Dystrophy Convenes Pulmonary Outcomes Workshop

Parent Project Muscular Dystrophy (PPMD) will convene a Pulmonary Outcomes Workshop in Bethesda, Maryland April 14-15. Leading physicians in the Duchenne pulmonary space, along with industry partners…


Added by PPMD on April 14, 2016 at 2:26pm — No Comments

PPMD’s Patient Preference Study about Pulmonary Outcomes – What we learned and why it matters

Late in 2015, Parent Project Muscular Dystrophy (PPMD) and our research partners at Johns Hopkins Bloomberg School of Public Health (JHSPH) released the results of our second patient-centered…


Added by PPMD on April 13, 2016 at 4:00pm — No Comments

Catabasis Pharmaceuticals Initiates Part B of the MoveDMD Trial of CAT-1004 for Duchenne

Catabasis announced positive biomarker data from Part A of the MoveDMD trial, as well as initiation of dosing for the first patient for Part B (Phase 2) - a 12-week trial to assess the efficacy of CAT-1004 in Duchenne. We look forward to continued success from our partners at…


Added by PPMD on April 12, 2016 at 9:30am — No Comments

PPMD's 2016 Connect Conference Agenda Now Available!

PPMD is excited to unveil the agenda for the 22nd Annual Connect Conference in Orlando, June 26-29 at…


Added by PPMD on April 11, 2016 at 1:16pm — No Comments

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