In a corporate update released today, PTC Therapeutics announced next steps for their drug, Translarna in the U.S. According to the release, PTC plans to file the Translarna New Drug Application (NDA) for nmDMD over protest with the U.S. FDA in…Continue
Added by PPMD on January 9, 2017 at 10:30am — No Comments
Today, PPMD announced a $2.2 million dollar grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PI), and Nationwide Children’s Hospital. Our purpose in awarding this grant is to open and accelerate the field of gene therapy – a strategy that seemed impossible as a potential…Continue
Added by Pat Furlong on January 6, 2017 at 9:00am — No Comments
We not only met our goal, we surpassed it!
We could not be more overwhelmed by the incredible response to our holiday campaign. In many ways, with a therapeutic pipeline full of promising treatments, it feels like the future of Duchenne therapy is here. Thanks to this community, we head into 2017 with…
Added by Pat Furlong on January 4, 2017 at 3:47pm — No Comments
Viral gastrointestinal (GI) viruses are no fun for anyone, but they are especially worrisome for a person living with Duchenne muscular dystrophy. GI viruses affect the GI track – the stomach and intestine (i.e., ‘gut’) – resulting in abdominal pain/discomfort, nausea, vomiting, intestinal…Continue
Added by Kathi Kinnett on January 4, 2017 at 10:30am — No Comments
We’re so close to our goal to fund CRISPR/Cas9!…Continue
Added by PPMD on December 29, 2016 at 9:30am — No Comments
Your donation will be DOUBLED. Now is the moment to give.…Continue
Added by PPMD on December 22, 2016 at 9:12am — No Comments
Help us raise $250,000 by 12/31!…
Added by Abby Bronson on December 20, 2016 at 12:00pm — No Comments
We have all had frustrations getting healthcare paid for, whether it is getting access and coverage for appointments, procedures, equipment, and/or medications.
Coverage is especially difficult when new medicines or procedures are recommended. Parents,…Continue
Help us raise $250,000 to fund CRISPR/Cas9.
After a few months of relative quiet, noise has returned to the Killian house in full force! For the holidays, all five kids are back under one roof with a revolving door of friends and endless groceries. Even with all the chaos that entails, this is my favorite time of…
Added by PPMD on December 13, 2016 at 9:38am — No Comments
When PPMD convened the Contemporary Issues in Duchenne Cardiology meeting with National Heart, Lung, and Blood Institute (NHLBI) in July 2014, there were several recommendations.
The strongest recommendations included the use of cardiac…Continue
Added by Kathi Kinnett on December 8, 2016 at 4:00pm — No Comments
It is the holidays – time for giving and laughter and hope. Hope comes in many forms, but I would imagine that for many reading this blog, hope comes in the form of a cure for Duchenne.
Could CRISPR/Cas9 offer hope for treating Duchenne?
As background, CRISPR/Cas9 is a…Continue
Added by Abby Bronson on December 8, 2016 at 3:30pm — No Comments
When I joined PPMD in 2012, care for Duchenne was all over the map.
Sadly, there were over 150 clinics spread across the US, but there was no oversight of these clinics – the community did not know who was providing care, the level of care being provided or whether anyone at…Continue
Added by Kathi Kinnett on December 8, 2016 at 3:02pm — No Comments
The Duchenne Community’s Voice Was Essential Every Step of the Way & Contains PPMD’s Provision to Strengthen Patient Focused Drug Development Efforts
To everyone in our PPMD community who called, reached out, emailed, and met with your elected…
Added by Annie Kennedy on December 7, 2016 at 2:30pm — No Comments
Duke University marked our final stop on PPMD’s 2016 Every Single One Tour, held on November 2. We were thrilled to be holding another tour stop at one of…Continue
Added by Ryan Fischer on December 7, 2016 at 11:04am — No Comments
We need you to call your two US Senators TODAY with one simple message – VOTE YES ON 21st CENTURY CURES!!
With just days remaining in the current Congress, this bill represents the extraordinary efforts of our PPMD community’s work and outreach with…Continue
Added by Ryan Fischer on December 5, 2016 at 10:07am — No Comments
We have so much momentum. Let’s seize this moment.
First of all, let me say THANK YOU! Your gift to PPMD on #GivingTuesday helped us surpass our goal, and we could not be more grateful. It is a wonderful way to kick off our holiday season—and we wanted to make sure you were among the…
Summit Therapeutics plc announced today that it has enrolled its first patients at trial sites in the US into PhaseOut DMD, a Phase 2 clinical trial of ezutromid in Duchenne. The 48-week, open-label (meaning all participants receive ezutromid) Phase 2 trial is enrolling up to 40 boys total between their 5th…Continue
Added by PPMD on November 16, 2016 at 9:00am — No Comments
Frequently, I get calls from mothers/sisters/aunts of people living with Duchenne about issues related to being a carrier of Duchenne:
PTC Therapeutics, Inc. today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna(TM) (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients…Continue
Added by PPMD on November 11, 2016 at 9:30am — No Comments