We finally have some very promising news for the development of adequate treatment for our boys with mutations requiring double or multi exon skipping!

In reading through this article about injecting a fully functional version of the dystrophin gene into the body using a harmless virus, it doesn't mention anything about applicability of multi-…

There is no way around it, today was hard. Hundreds of us – the largest gathering of Duchenne families in history – came together to tell the FDA that eteplirsen is safe, effective, and should be approved using the tools that have been given to the FDA, tools that our community worked…

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…

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…

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 the…

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…

On Saturday, Duchenne community members from six states gathered together at the beautiful facilities at Kennedy Krieger in Baltimore – one of PPMD’s Certified Duchenne Care Centers – to hear the latest developments on clinical trials, clinical care practices,…

Bipartisan Group of Senators Urge FDA to Utilize All Available Tools to Speed Access to Promising Duchenne Therapies

On Friday April 15th, U.S. Senators Roger Wicker, (R-MS) and Amy Klobuchar, (D-MN), sent a…

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

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…

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…

Earlier this year, 109 members of the House of Representatives sent a powerful letter to the FDA urging them to “utilize all available tools, resources, and authorities” in reviewing a treatment for Duchenne muscular dystrophy. Congressional champions Congressmen Mike Fitzpatrick (R-PA), Bill Keating (D-MA), and Peter King (R-NY) led this incredible bipartisan letter.…

We are just a few short weeks from the FDA Advisory Committee Meeting for eteplirsen on April 25th. PPMD is part of a united coalition of Duchenne organizations (MakeDuchenneHistory Coalition) working together to ensure we make the largest possible impact on the advisory committee and the…