April 2015 Blog Posts (10)


Hi Dr.Porter,
Welcome to PPMD! This is a good start to Robotics.
Could you give us a time-line when you think these modifications matched just for duchenne boys will come to fruition?
One never knows just how quickly these nooks & crannies can be worked out when you're in a room filled with brilliant minds!

Added by Judith Counterman on April 30, 2015 at 4:27pm — No Comments

The “Let’s Get Them Moving” Workshop

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…


Added by PPMD on April 29, 2015 at 10:00am — 1 Comment

Let's Get Them Moving Workshop: Our First Steps Toward That Goal

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

reflecting back to the advocacy conference/PAAC initial meeting

  Attending the initial meeting of the PAAC in DC this last February was quite an amazing experience. It was our first live get together. I was so very impressed by the knowledge and enthusiasm of the group. We started out having 2 days of meetings and presentations. At he end of those two rather intense days we came together with a consensus of the things we as a committee were going to be tackling.

  One of the things that the group was asked to do was to come up with someone from…


Added by michael stone on April 27, 2015 at 3:13pm — 1 Comment

A Light Goes Out – Goodbye to Ben Cumbo, PAAC Member

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 each. He asked Senator Spector if he was willing to put…


Added by Pat Furlong on April 22, 2015 at 3:30pm — 4 Comments

Santhera Releases Positive Data from Phase III Clinical Trial of Idebenone (Raxone®/Catena®) in Duchenne

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…


Added by John D. Porter, PhD on April 21, 2015 at 10:00am — No Comments

Parent Project Muscular Dystrophy Awards $148,000 Grant to University of Washington

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…


Added by PPMD on April 20, 2015 at 11:00am — No Comments

BioMarin Update

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.

BioMarin Update on Duchenne Muscular Dystrophy…


Added by Ryan Fischer on April 14, 2015 at 9:00am — No Comments

Sarepta Update

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 with Duchenne patient organizations and foundations to update them…


Added by Ryan Fischer on April 13, 2015 at 11:00am — No Comments

Orthopedic Complications in Duchenne (Webinar Summary & Recording)

"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 asked regularly around orthopedic complications in Duchenne.…


Added by Kathi Kinnett on April 3, 2015 at 12:00pm — No Comments

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