PPMD applauds the group of 83 international experts with experience caring for Duchenne patients around the globe who have written a letter to the FDA encouraging approval of ataluren. With hundreds of patients accessing ataluren through…Continue
Added by PPMD on October 9, 2017 at 10:36am — No Comments
Annie Kennedy and Kathi Kinnett from the PPMD team are attending the 22nd International Annual Congress of the World Muscle Society and will be reporting back over the next couple of days. More…Continue
Added by PPMD on October 6, 2017 at 1:30pm — No Comments
Catabasis Pharmaceuticals, Inc. today reported new positive efficacy results following 24 and 36 weeks of treatment with 100 mg/kg/day oral edasalonexent in the MoveDMD Phase 2 trial open-label…Continue
Added by PPMD on October 3, 2017 at 9:30am — No Comments
On Thursday, September 28, an FDA Advisory Committee will review ataluren for potential approval and use within our Duchenne community.
The Duchenne community is joining forces in a show of strength to…
Added by PPMD on September 27, 2017 at 6:42pm — No Comments
PPMD is excited to announce that we will continue a grant program to help support our Certified Duchenne Care Center (CDCC) Program. The CDCC Program is a network of…
Added by PPMD on September 27, 2017 at 12:30pm — No Comments
This weekend, nearly every televised College Football game around the country will have coaches wearing Coach To Cure MD armbands, tweeting about Duchenne, and encouraging fans to text a…Continue
Added by PPMD on September 26, 2017 at 1:00pm — No Comments
Added by PPMD on September 26, 2017 at 9:30am — No Comments
Santhera Pharmaceuticals announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion for its Type II extension application for Raxone® (idebenone) in Duchenne. Santhera plans to appeal the opinion and will seek a re-examination by the CHMP. We appreciate…Continue
Added by PPMD on September 15, 2017 at 8:00am — No Comments
Today Parent Project Muscular Dystrophy (PPMD) named Nemours/Alfred I duPont Hospital for Children (Nemours) in Wilmington, Delaware our 17th Certified Duchenne Care Center.
PPMD’s Certified Duchenne Care Center Program, which supports standardized, comprehensive care and services for all…Continue
Added by PPMD on September 14, 2017 at 8:30pm — No Comments
The deadline to submit a written comment for the FDA Advisory Committee Meeting on ataluren is this upcoming Thursday, September 14th.
To make a comment,…Continue
Added by PPMD on September 12, 2017 at 9:30am — No Comments
PPMD is excited to announce next year’s lineup of areas we will visit as part of our End Duchenne Tour!
Combining each of the pillars that make up PPMD’s mission, the End Duchenne Tour brings updates on research, advocacy, and care to cities across the country, featuring a roster of leading experts in the…
Added by PPMD on September 8, 2017 at 5:30pm — No Comments
Added by PPMD on September 6, 2017 at 10:00am — No Comments
Earlier this year, Bristol-Myers Squibb (BMS) announced it had entered into an agreement to license BMS-986089 (RG6206), an anti-myostatin adnectin in development for Duchenne muscular dystrophy, to Roche. Today, the Roche team has provided an update to our…Continue
Added by PPMD on September 5, 2017 at 9:30am — No Comments
The Duchenne community is now preparing for the September 28 Advisory Committee Meeting for PTC’s therapy ataluren. Whether submitting written testimony and/or attending the Ad Comm in person, there are multiple…Continue
Added by PPMD on August 22, 2017 at 11:00am — No Comments
Today Parent Project Muscular Dystrophy (PPMD) named Gillette Children's Specialty Healthcare Neuromuscular Clinic (Gillette) our 16th Certified Duchenne…Continue
Added by PPMD on July 13, 2017 at 11:21am — No Comments
Added by PPMD on June 30, 2017 at 1:49pm — No Comments
Today, PPMD is convening medical experts from around the world for a workshop exploring inflammation and immunity in Duchenne on the eve of our 23rd Annual Connect Conference.
In January, PPMD organized a Critical Path Innovation Meeting with the FDA focused on…Continue
Added by PPMD on June 28, 2017 at 2:19pm — No Comments
Recently, FDA Commissioner Scott Gottlieb announced that the hiring freeze, instituted on January 23, has been lifted.
This reversal means that the FDA can begin to fill vacancies – needed resources for continued research into rare disease treatments and for the review and approval of new medicines.
PPMD is proud to have been one…Continue
Added by PPMD on June 15, 2017 at 11:53am — No Comments
Once again, PPMD has signed on to letters to leaders in both the House and Senate, requesting they continue to support the critical and highly successful defense health research programs funded through the Congressionally Directed Medical Research Programs (CDMRP) at the Department of Defense (DoD) for FY 2018.…Continue
Added by PPMD on June 15, 2017 at 11:30am — No Comments