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-…
ContinueAdded by Jeffrey Kopp on April 27, 2016 at 3:37pm — No Comments
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…
ContinueAdded by Pat Furlong on April 25, 2016 at 10:30pm — 5 Comments
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…
ContinueAdded by PPMD on April 21, 2016 at 8:30am — No Comments
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…
ContinueAdded by PPMD on April 21, 2016 at 8:00am — No Comments
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…
ContinueAdded by PPMD on April 19, 2016 at 4:00pm — No Comments
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…
ContinueAdded by PPMD on April 19, 2016 at 9:00am — No Comments
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,…
ContinueAdded by Annie Kennedy on April 18, 2016 at 12:30pm — No Comments
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…
ContinueAdded by Ryan Fischer on April 18, 2016 at 9:44am — No Comments
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…
ContinueAdded by PPMD on April 15, 2016 at 10:30am — No Comments
Added by PPMD on April 14, 2016 at 2:26pm — No Comments
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…
ContinueAdded by PPMD on April 13, 2016 at 4:00pm — No Comments
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…
ContinueAdded by PPMD on April 12, 2016 at 9:30am — No Comments
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
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.…
ContinueAdded by Ryan Fischer on April 5, 2016 at 11:30am — No Comments
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…
ContinueAdded by Ryan Fischer on April 4, 2016 at 10:30am — 1 Comment
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