With this year's Annual Connect Conference in Washington, DC, PPMD wanted to take advantage of being in our Nation's Capital and ensure that our Duchenne community leaves an indelible imprint…Continue
Added by PPMD on May 19, 2015 at 12:30pm — No Comments
For 21 years, PPMD has been connecting the Duchenne community with experts in the field focused on research, advocacy, and care. Our largest gathering is, of course, PPMD’s Annual Connect…Continue
Added by PPMD on May 12, 2015 at 11:00am — No Comments
Last night, another beautiful spirit in the Duchenne community left this world much too early. Matt Petrusko was a 34-year-old from Rahway, New Jersey and a proud member of PPMD’s…Continue
As you know, PPMD works very hard to keep you as up to date as possible. Sometimes those updates come very quickly, as has recently happened with recommendations for vaccinations. While we just recently published a new “altered vaccination schedule,” discussions with the Centers for Disease Control and…Continue
Added by Kathi Kinnett on May 7, 2015 at 1:30pm — No Comments
PPMD is excited to share with you the agenda for the 21st Annual Connect Conference in Washington, D.C., June 18-21 at the Washington Marriott Wardman Park. Every year our agenda grows and…Continue
Added by PPMD on May 4, 2015 at 5:00pm — No Comments
On Monday, PPMD’s Founding President Pat Furlong, CEO Dr. John Porter, and SVP of Legislation & Public Policy Annie Kennedy joined Duchenne stakeholders from around the world in a special one-day meeting to discuss robotic technology and the potential it holds for our community. PPMD – together with UPPMD – gathered the best and brightest robotics experts and engineers who are working to apply their innovations to Duchenne. We discussed the variety of technologies currently in…Continue
Yesterday, Parent Project Muscular Dystrophy and Duchenne Parent Project-Netherlands (DPP) co-hosted the first ever conference on assistive technology for Duchenne muscular dystrophy. There were twenty-five participants in the workshop, from six countries, representing academia (both researchers and engineers), companies, clinical practice, and patient advocacy. PPMD was represented by Pat Furlong, Annie Kennedy, and myself. Elizabeth Vroom (DPP-Netherlands) set the tone for the meeting…
Added by John D. Porter, PhD on April 28, 2015 at 10:00am — No Comments
Ben Cumbo died last night. While Heaven is brighter today, our world has lost a great light.
I first met Ben in 2001. We testified about the importance of the MD-CARE Act. He was very young then, but his wisdom apparent. He talked about the cost of a Stealth bomber, on average $737 million…
Today, Santhera has published the results in the highly prestigious journal, The Lancet, from their successful clinical trial of Raxone®/Catena® . They will present these results later this week, at the annual meeting of the American Academy of Neurology. First, idebenone was safe and well tolerated. Importantly, treatment of study…Continue
Added by John D. Porter, PhD on April 21, 2015 at 10:00am — No Comments
Parent Project Muscular Dystrophy (PPMD) has awarded University of Washington a $148,000 grant to continue the functional analysis of spectrin-like repeats in dystrophin. Led by Stanley C. Froehner, PhD, Sackler professor and chair of the Department of Physiology & Biophysics at University of Washington, this grant will provide funding so that more research can be completed before the team submits to the National Institutes of Health (NIH) for additional…Continue
Added by PPMD on April 20, 2015 at 11:00am — No Comments
BioMarin has provided the following update on redosing of drisapersen for a new clinical trial. Ambulation will not be an inclusion or exclusion criterion for enrollment, but boys will need to have been previously dosed as part of a clinical trial.…
Added by Ryan Fischer on April 14, 2015 at 9:00am — No Comments
Earlier this month Sarepta announced a change in leadership, with CMO Ed Kaye taking over as interim CEO.
Following that announcement the team at Sarepta organized a call…
Added by Ryan Fischer on April 13, 2015 at 11:00am — No Comments
"What is heel cord lengthening?"
"What are AFO's?"
"How do I know if I am correctly doing stretches with my child?"
"What should I know about scoliosis surgery?"
"My child hates "night boots" – are they really that important?"
These and so many other questions are…Continue
Added by Kathi Kinnett on April 3, 2015 at 12:00pm — No Comments
I always felt a strong connection to the Duchenne community while working at the NIH. As you know, that connection and the incredible work of organizations like PPMD inspired me to jump full-time into the fight to end Duchenne.
Yesterday’s FDA and NIH dystrophin methodology workshop really…Continue
John Porter and I have been invited to participate in the FDA-NIH dystrophin methodology meeting on Friday, March 20. While we said yes, we realize this is a difficult and painful topic for all of us. Dystrophin feels like the brass…Continue
Added by Pat Furlong on March 18, 2015 at 9:30am — No Comments
On January 15 of this year we introduced you to an incredibly special group of people, PPMD’s Adult Advisory Committee. The PPMD Adult Advisory Council (PAAC) serves to ensure that the…Continue
Added by Annie Kennedy on March 16, 2015 at 11:00pm — No Comments
PPMD loves creative, out-of-the-box fundraisers that raise awareness. This new, annual event from Davenport, FL is a great example of something you can do in your community. Read organizer Diana Rodriguez’s story and don’t hesitate to reach out to me with your own ideas. PPMD has all the tools you need to create a truly memorable and important event!…Continue
Added by Danielle Garrigan on March 16, 2015 at 8:34pm — No Comments
Despite great odds in a political climate that was stacked against us – we achieved what was considered to have been impossible. After months of outreach to congressional champions and visits to Capitol Hill, the…Continue
Added by Annie Kennedy on March 15, 2015 at 12:04pm — No Comments
Epicatechin is a flavonoid found in dark chocolate harvested from the cacao tree. What’s that got to do with Duchenne? Epicatechin is one of dozens of flavonoids found in dark chocolate, but it is unique with respect to its effects on muscle and heart. Epicatechin has been demonstrated in animals and…Continue
[NOTE: This blog has been updated. Please see below for more information.]
PPMD has been in touch with Masters Pharmaceuticals regarding a recall that was sent…Continue