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…Continue
Added by Ryan Fischer on October 20, 2016 at 1:00pm — 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…Continue
Added 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…
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…Continue
Added 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…Continue
Added 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!…Continue
Added by PPMD on October 14, 2016 at 9:57am — 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…
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…Continue
Added 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…Continue
Added 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 –…Continue
Added 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…Continue
Added 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.…Continue
Added 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…
Added by Ann Martin on October 5, 2016 at 1:00pm — No Comments
Catabasis announced that target enrollment of 30 patients has been reached for Part B of the MoveDMD trial, a 12-week trial to assess the safety and efficacy of edasalonexent (CAT-1004) in Duchenne. Edasalonexent is an oral small molecule that the company believes has the potential to be a…Continue
Added by PPMD on October 4, 2016 at 11:00am — No Comments
PTC Therapeutics announced that the Proceedings of the National Academy of Sciences (PNAS) has published new results further validating Translarna's™ (ataluren) mechanism of action to promote readthrough of premature stop codons resulting from nonsense mutations in…Continue
Added by PPMD on October 4, 2016 at 11:00am — No Comments
Today, Sarepta Therapeutics and Summit announced a license and collaboration agreement, which grants Sarepta the right to commercialize Summit's utrophin modulators in Europe should they receive approval. This includes ezutromid, which is currently in the Phase 2…Continue
Added by PPMD on October 4, 2016 at 8:30am — No Comments
PPMD is thrilled to learn of the collaboration of Catabasis and Sarepta Therapeutics, two partners deeply committed to this community. This joint research collaboration will…Continue
Added by PPMD on September 29, 2016 at 9:21am — No Comments
Sarepta Therapeutics today announced the first patient dosed in the phase III clinical trial of SRP-4045 and SRP-4053 in patients with Duchenne muscular dystrophy amenable to exon 45 or 53 skipping.
Read the announcement from Sarepta:…Continue
Added by PPMD on September 28, 2016 at 6:30pm — No Comments
Santhera Pharmaceuticals today announced that the first patient has been enrolled in Santhera's phase III (SIDEROS) trial. The Phase III SIDEROS trial is designed to confirm the efficacy of Raxone in patients…Continue
Added by PPMD on September 28, 2016 at 8:54am — No Comments
Summit Therapeutics plc today announced it has received Fast Track designation from the US Food and Drug Administration (FDA) for ezutromid in the treatment of…Continue