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,…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…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…
Added by PPMD on September 8, 2017 at 5:36pm — 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…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…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 …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…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…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…Continue
Added by PPMD on June 15, 2017 at 11:30am — No Comments
PPMD's Pat Furlong
Earlier this month, we had the privilege of having our fourth stop in 2017 on …Continue
Added by PPMD on June 14, 2017 at 10:30am — No Comments
PTC Therapeutics, Inc. today announced that the FDA has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna™). This is another important moment for our community and we look forward to the opportunity to share our collective experiences with Translarna with the FDA. As details on this Ad Comm become…Continue
Added by PPMD on June 6, 2017 at 8:47am — No Comments
Added by PPMD on June 2, 2017 at 11:30am — No Comments
This Mother’s Day, thank someone for their role in the fight to end Duchenne.
If you’re reading this blog, chances are you know my wife, Pat Furlong. For 23 years now, Pat has been leading Parent Project Muscular Dystrophy (PPMD) and the fight to end Duchenne. Many of you have told me over the years that Pat has been a source of comfort,…Continue
Added by PPMD on May 10, 2017 at 10:30am — No Comments
PPMD is excited to unveil the agenda for our 2017 Connect Conference in Chicago, June 29 - July 2, 2017!
We are so proud of the …Continue
Added by PPMD on May 9, 2017 at 11:00am — No Comments
Today, PTC Therapeutics provided a community update on EMFLAZA (deflazacort) which they acquired earlier in the year from Marathon Pharmaceuticals. The community has anxiously awaited an update on both access and pricing ever since the deal between PTC and Marathon was announced.
On today’s call, we…Continue
Added by PPMD on May 8, 2017 at 5:38pm — No Comments
PTC Therapeutics has released a letter to the community and will host a conference call for the Duchenne community to discuss the launch plans for EMFLAZA™ (deflazacort) on Monday, May 8, 2017 at 3:00 PM EDT (call-in details are below).
PTC Therapeutics, Inc. will host a conference call for the Duchenne community to discuss the launch plans for Emflaza™ (deflazacort) on Monday, May 8, 2017 at…Continue
Added by PPMD on April 26, 2017 at 10:00am — No Comments