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
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…
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…Continue
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
Today, my sons are dancing! Today we ALL celebrate the first FDA approval in Duchenne. Today the FDA approved Exondys 51 (aka eteplirsen) injection, specifically indicated for patients…Continue
For the third year in a row, the global Duchenne community will come together on September 7 for World Duchenne Awareness Day. This day, this simple idea, has blossomed into a truly incredible opportunity for families and friends…Continue
Added by Pat Furlong on August 31, 2016 at 2:30pm — No Comments
Last week you heard from my friend Kim Powers. She and her husband have jumped into this year’s …Continue
Added by Pat Furlong on August 10, 2016 at 11:30am — No Comments
PTC recently participated in discussions with FDA to discuss the Refuse to File (RTF) letter issued on February 22, 2016 with respect to the company's New Drug Application (NDA) for Translarna for the treatment of Duchenne. Today PTC…Continue
Added by Pat Furlong on July 25, 2016 at 12:00pm — No Comments
Today is June 1 and I know many of us feel like 2016 has beat up this community already. With every small victory, we face another major setback.
As you have heard by now,…Continue
I am a genetic carrier of Duchenne. My mother was not. This means I was one of the 1:4600 females born with this genetic mutation. As a child, I had muscle cramps, ‘growing pains’ my parents would say. And from those ‘growing pains,’ 20 years later, my sons were diagnosed and I first learned…Continue
There is no way around it, today was hard. Hundreds of us – the largest gathering of Duchenne families in history – came together to tell the FDA that eteplirsen is safe, effective, and should be approved using the tools…Continue
We are not a team because we work together. We are a team because we respect, trust, and care for each other. – Vala Afshar
Added by Pat Furlong on February 1, 2016 at 9:43am — No Comments
Today our community was dealt a painful blow as the FDA issued a Complete Response letter to BioMarin regarding the company’s New Drug Application (NDA) for Kyndrisa (drisapersen). As stated in BioMarin’s press release, “The FDA issues Complete Response letters to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form. FDA…Continue
Last month we told you we hoped to raise $200,000 to support new, early-stage research. Throughout the month, we told you about potential therapies like epicatechin. We told you about generous matches from Mary-Lou and Larry Weisman and the Dumm Family/…Continue
Added by Pat Furlong on January 5, 2016 at 11:00am — No Comments
This community should be proud of the role we have played over the last several weeks preparing for the potential approval of multiple therapies to treat Duchenne. As I mentioned last week, there is still…Continue
Added by Pat Furlong on November 30, 2015 at 3:00pm — No Comments
It has been an incredibly intense few months as we hit the homestretch of 2015 and head into a promising 2016. One Advisory Committee meeting is down and we have another quickly approaching most likely in late January 2016.…Continue
I have never been so proud of this community. Today our community reached a historic milestone, a moment I wondered if we would ever see. Today’s FDA Advisory Committee (Ad Comm) for BioMarin’s drisapersen, gave us an…Continue
After yesterday’s news regarding the Ad Comms for both BioMarin and Sarepta, the community continues to ask questions. PPMD has been working around the clock to get some answers…Continue
Added by Pat Furlong on October 15, 2015 at 3:30pm — No Comments