As we head into the 10th Annual Coach To Cure MD weekend tomorrow, with emotions still running high from yesterday’s…
Added by Pat Furlong on September 29, 2017 at 10:06am — No Comments
Like any of you who participated in today’s Advisory Committee Meeting at the FDA for PTC Therapeutic’s ataluren, I am emotionally exhausted. Whether you participated by speaking during the Open Public Hearing, attending the Ad Comm on the FDA campus, submitting your written testimony, or streaming the meeting from home – I would imagine that you, too, are…Continue
Added by Pat Furlong on September 28, 2017 at 5:30pm — 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
Let’s say that Joe has Duchenne. If Joe breaks his arm and the EMT comes before Joe’s parent arrives, Joe may not remember to tell the EMT that: 1) he has Duchenne, 2) he is on daily steroids 3) he is allergic to penicillin and, in that painful scary moment, he may or may not remember his parent’s cell phone number.
For some people, having a medical ID that…Continue
Added by Kathi Kinnett on September 22, 2017 at 7:23pm — No Comments
As you know by now, September is Duchenne Action Month. And while the idea behind this campaign is to encourage our community to take…Continue
Added by Abby Bronson on September 21, 2017 at 12:00pm — No Comments
The Senate is considering another effort to repeal the Affordable Care Act (ACA) before the end of the month. Last week, Senators Lindsey Graham (R-SC), Bill Cassidy (R-LA), Dean Heller (R-NV), and Ron Johnson (R-WI) released a new plan to repeal and replace the ACA. This proposal is commonly referred to as…Continue
Added by Annie Kennedy on September 19, 2017 at 10:00am — No Comments
Everyone, six months of age and older, needs to be vaccinated against influenza (flu) by the end of October, if possible. Protecting people living with Duchenne is especially important. A 2005 study done by the…Continue
Added by Kathi Kinnett on September 18, 2017 at 10:30am — 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 people…Continue
Added by PPMD on September 14, 2017 at 8:30pm — No Comments
There are some days when I feel like our community is in its infancy. When I look at where we are in terms of access, therapeutic options, combinations, care implementation…
But more often than not, I am overwhelmed with gratitude for the infrastructure and resources available to our Duchenne community. The rich collaborations and strong…
Added by Annie Kennedy on September 13, 2017 at 5:00pm — No Comments
We kicked off our fall Every Single One Tour stops in West Virginia this past weekend! We appreciate all of the families, speakers, and volunteers that spent the day with us in Charleston. …Continue
Added by Kathi Kinnett on September 13, 2017 at 4:30pm — No Comments
Paperwork – oh the paperwork!
Each time you visit a physician’s office or hospital, we leave with piles of forms and papers detailing the visit. The providers seen, the prescriptions written, results from tests conducted, instructions for follow up, and more. And each of those pieces of paper includes numerous codes that allow for tracking…Continue
Added by Annie Kennedy on September 13, 2017 at 10:00am — 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
The dynamics of weight loss are intriguing, in take a look at the many because they came from desire to shed some weight in comparison to the small number who do. You would think with all the diet plans and dietary supplements, that weight problems would go away. But that hasn't came about, so something must be wrong. The sheer number of programs and products out there helps it to be difficult to find out what is effective and what is not. And it is far from as if one solution is effective…Continue
Added by Louie Lewis on September 11, 2017 at 9:56am — 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 Duchenne…
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