In an effort to answer some of the questions that have arisen with the potential FDA approval of deflazacort in the U.S., PPMD has worked with both Masters and Marathon to put together the resource below, which we hope will provide clarity.
Webinar…
ContinueAdded by Kathi Kinnett on October 31, 2016 at 2:00pm — No Comments
Patient Assistance programs are typically non-profit organizations sponsored by drug companies, patient advocacy organizations, and civic groups that establish disease specific programs to facilitate access to brand-name medications.
In the case of…
Added by Annie Kennedy on October 28, 2016 at 11:00am — No Comments
PPMD Awards Twelfth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care
The Center for Duchenne Muscular Dystrophy at the University of California, Los Angeles…
ContinueAdded by PPMD on October 26, 2016 at 3:02pm — No Comments
PPMD Awards Twelfth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care
The Center for Duchenne Muscular Dystrophy at the University of California, Los Angeles…
ContinueAdded by PPMD on October 26, 2016 at 3:02pm — No Comments
The journey has been long. From PTC Therapeutics' first presentation about targeting a ‘stop sign’ within the dystrophin mutation to PTC 124 to ataluren and now, Translarna. The journey has been a long one.
The team at PTC are pioneers, the company…
ContinueAdded by Pat Furlong on October 26, 2016 at 12:30pm — 1 Comment
Solid Biosciences and its subsidiary, Solid GT, announced today that the FDA and the European Commission have granted Orphan Drug designations for the company’s gene therapy candidate, SGT-001,…
ContinueAdded by PPMD on October 24, 2016 at 9:00am — No Comments
You are invited to join us February 12-14, 2017 in Washington D.C. for what is going to be another critical year in Duchenne advocacy.
2016 marked a tremendous…
ContinueAdded by Ryan Fischer on October 20, 2016 at 1:00pm — No Comments
This presentation, "The Emergent and Acute Management of Patients Living with Duchenne Muscular Dystrophy," was produced in an effort to educate clinicians in the care of individuals and families living with Duchenne.
The presentation can be…
ContinueAdded by Kathi Kinnett on October 19, 2016 at 3:30pm — No Comments
When I first joined the Duchenne community in 2009 at Children’s National Medical Center, I remember thinking that I was walking into a critical juncture in Duchenne history. There were three compounds in the clinic (drisapersen, ataluren, and eteplirsen aka Exondys 51) with more waiting in the wings to go into clinical trials, a drug…
ContinueAdded by Abby Bronson on October 18, 2016 at 1:00pm — No Comments
Join us for our first DuchenneConnect Twitter chat on Thursday, Oct 20, 2016 from 1-2:00 PM EST. We will be using the hashtag #DuchenneConnectTC.
Our first chat will cover the importance of PPMD's patient registry,…
Added by Ann Lucas on October 17, 2016 at 11:30am — No Comments
PPMD is disappointed to learn that the FDA denied PTC Therapeutics' first appeal of the refuse to file letter issued in February. As we understand it, this is a process and may involve additional appeals. PPMD will be…
ContinueAdded by PPMD on October 17, 2016 at 10:24am — No Comments
It is hard to imagine anything worse than receiving the diagnosis of Duchenne. The dates and odysseys that brought us into this community differ, but the fervor we share to slow and stop the progression of this disease as it marches through our children is unparalleled in any community.
But today I know that there actually is something worse…
ContinueAdded by Pat Furlong on October 14, 2016 at 12:02pm — No Comments
Can you believe 2017 is just around the corner? It will be here before you know it and PPMD is busy putting together an exciting list of events that will continue to connect and inform our Duchenne community. It’s already a packed new year!
Check out where your PPMD family plans to be in 2017 and mark your calendars!…
ContinueAdded by PPMD on October 14, 2016 at 9:30am — No Comments
With the initiation of 4 states ABLE programs already begun and Oregon having just anounced that their program will launch in December, any qualified individual (meaning most families with Duchenne) can open their accounts in 2016, if interested. We have previously shared information about the ABLE program and the…
Added by Annie Kennedy on October 13, 2016 at 8:37pm — No Comments
Parent Project Muscular Dystrophy (PPMD) has awarded a $239,000 grant to Dr. Terence Partridge, Professor of Systematic Integrative Biology, Research Center for Genetic Medicine at Children's National Medical Center. This grant will help Dr. Partridge and his team continue their work in understanding exon skipping technology for future trials in…
ContinueAdded by PPMD on October 13, 2016 at 11:31am — No Comments
Ever wished you could change the world – or at least your piece of it? Been looking for a way to engage a bit more? Have personal experience that you’d like to see be better reflected in national policy and program efforts?
PPMD is seeking adult members of our Duchenne/Becker community to serve on the PPMD Adult Advisory Committee…
ContinueAdded by Annie Kennedy on October 12, 2016 at 11:30am — No Comments
Last week, PPMD began a webinar series on access – an exciting new chapter for our Duchenne community. But we are just at the beginning of this chapter and there is lots to explore.
We knew this day was coming – we knew that one day we would have our…
ContinueAdded by Annie Kennedy on October 11, 2016 at 3:00pm — No Comments
PPMD is deeply troubled to learn that health insurer Anthem will not be covering Exondys 51, stating the following position:
“The use of eteplirsen is considered investigational and not medically necessary for all indications, including but not limited to the treatment of Duchenne muscular dystrophy.”
This is not an…
ContinueAdded by PPMD on October 7, 2016 at 2:30pm — No Comments
PTC Therapeutics, Inc. today announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory people with nonsense mutation Duchenne. PPMD continues to urge the FDA to provide PTC with a full review and advisory committee meeting.…
ContinueAdded by PPMD on October 6, 2016 at 9:15am — No Comments
Last week, Ann Lucas and I had the honor of presenting an Educational Breakout Session focused on Duchenne at the National Society of Genetic Counselors (NSGC) Annual Education Conference in Seattle. Lauren Morgenroth from TRiNDS also presented during the session, as well as Laurie Paschal, a Duchenne mom living in…
Added by Ann Martin on October 5, 2016 at 1:00pm — No Comments
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