PPMD's Pat Furlong
Earlier this month, we had the privilege of having our fourth stop in 2017 on PPMD's Every Single One Tour in Gainesville,…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
Update from PTC…Continue
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 3:00pm (ET).
The call can be…Continue
Added by PPMD on April 26, 2017 at 10:00am — No Comments
Capricor Therapeutics today announced positive top-line results from a safety and exploratory efficacy analysis of six-month data from the randomized 12-month Phase I/II HOPE Clinical Trial of CAP-1002 (allogeneic cardiosphere-derived cells), an investigational candidate for the treatment of patients with Duchenne.
We are excited to see these results and to see the field of cell…Continue
On May 18, 2017, FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s…Continue
Added by PPMD on April 24, 2017 at 12:30pm — No Comments
Today, PTC Therapeutics announced that they have completed their acquisition of Emflaza™ (deflazacort) ahead of schedule, following early conclusion of the anti-trust review.
PTC will be hosting a call with the Duchenne community the week of May 8 to provide details on the plan for the launch of Emflaza. We look forward to learning more from PTC in the…Continue
Added by PPMD on April 20, 2017 at 4:00pm — No Comments
Bristol-Myers Squibb (BMS) today announced that it has entered into an agreement to license BMS-986089, an anti-myostatin adnectin in development for Duchenne muscular dystrophy, to Roche. …Continue
Added by PPMD on April 13, 2017 at 10:03am — No Comments
Iowa Children’s Hospital marked our third stop on PPMD’s 2017 Every Single One Tour, held on April 1, 2017. The amazing staff at Iowa Children’s Hospital helped make this a wonderful day and we can see why they are one of the 15 …Continue
Added by PPMD on April 7, 2017 at 1:30pm — No Comments
Today Parent Project Muscular Dystrophy (PPMD) named UC Davis Department of Physical Medicine and Rehabilitation Neuromuscular Disease Clinic (UC Davis) our 15th …Continue
Added by PPMD on April 6, 2017 at 12:30pm — No Comments
The PPMD Every Single [One] Tour had an amazing turnout last weekend in Seattle, WA! Nearly 100 parents, patients and professionals gathered at Seattle Children’s Hospital to talk and learn about research, care, therapy, and clinical…Continue
Added by PPMD on March 31, 2017 at 11:30am — No Comments
Summit Therapeutics announced today that it will proceed with the planned extension phase of PhaseOut DMD, a Phase 2 clinical trial evaluating the utrophin modulator ezutromid, subject to regulatory approval.
This follows an interim review of the safety and tolerability data from the ongoing trial by an independent Data…Continue
Added by PPMD on March 27, 2017 at 11:25am — No Comments
Health insurance is a contract between you and an insurance company. Health insurance helps pay your medical bills when you need care.
There are two major types of health insurance:
Today ReveraGen BioPharma Inc, announced that the FDA has granted Fast Track designation for vamorolone (VBP15) for the treatment of Duchenne. This designation can speed the review of efficacy and safety data for vamorolone in people with Duchenne, potentially leading to more rapid regulatory approval. PPMD is pleased to learn this news and happy to have provided…Continue
Added by PPMD on March 24, 2017 at 12:30pm — No Comments
Today, PTC Therapeutics provided the following update to the Duchenne community regarding their acquisition of Emflaza™ (deflazacort) from Marathon Pharmaceuticals. We appreciate PTC keeping our community updated during this transition.
March 24, 2017
Added by PPMD on March 24, 2017 at 10:38am — No Comments
PPMD is pleased to learn that the FDA has completed its review and concluded that Akashi Therapeutics may resume clinical development of HT-100 (delayed-release halofuginone) in people with Duchenne (click here to read Akashi's press release). Preventing fibrosis is an important target and an essential piece of a combination of therapies that will be…Continue
Added by PPMD on March 22, 2017 at 9:00am — No Comments