Parent Project Muscular Dystrophy (PPMD) has awarded University of Minnesota researcher Dr. Rita Perlingeiro a grant for $220,000 to develop a therapeutic approach for Duchenne using a new type of "adult-derived" stem cell called "induced pluripotent stem cells" or "iPSCs." iPSCs can be created from non-controversial sources like skin, have the ability to multiply indefinitely, and can be coaxed to become many different kinds of tissue types.
When Chris and Patrick died, there was no Internet, no way to connect. The days just after they died, life was busy but at the same time heartbreaking – there was so little time to think about anything. After all was over, silence settled in. We were left with the burden of living without them, of going on and trying to make some sense of it.
No one called, not…Continue
Added by Ryan Fischer on March 7, 2013 at 4:21pm — No Comments
Updated 3/07/14: 109 total members- New additions in bold belowContinue
Added by Ryan Fischer on March 5, 2013 at 3:00pm — No Comments
PPMD will provide $100,000 to Dr. Stanley Nelson of the University of California, Los Angeles, to examine the genomes of 40 boys with Duchenne who are either very mildly affected or very severely affected. The goal is to identify changes in genes other than dystrophin that may affect the course of the disease.
"We know that even boys who have exactly the same mutation in the dystrophin…
Added by PPMD on March 5, 2013 at 10:49am — No Comments
The Muscular Dystrophy Association, Parent Project Muscular Dystrophy and the Foundation to Eradicate Duchenne have called on the U.S. Senate and the U.S. House of Representatives to reauthorize the MD CARE Act of 2001, and to continue federal support for the accelerated pace of research and treatment development for muscular dystrophy. We are urging the public to contact their U.S. Senators and ask them to vote for reauthorization of …Continue
Added by PPMD on February 19, 2013 at 10:30am — No Comments
As you know, we head into an important year of Advocacy with the muscular dystrophy community determined to Reauthorize the MD-CARE Act. For the past few months PPMD, FED, and MDA have been working…Continue
Added by Ryan Fischer on January 28, 2013 at 4:30pm — No Comments
Added by PPMD on January 7, 2013 at 10:30am — No Comments
The 194th workshop of the European Neuro Muscular Centre (ENMC) met in Naarden, The Netherlands, December 7-9, 2012. The title of the workshop was, “Towards Clinical Application of Antisense-mediated Exon Skipping for Duchenne Muscular Dystrophy – Opportunities and Challenges.” 25 representatives of…Continue
Added by Kathi Kinnett on December 13, 2012 at 12:30pm — No Comments
Added by Pat Furlong on December 13, 2012 at 10:30am — No Comments
This year is no exception. We’ve accomplished so much together. When I think of all that our community has made possible over the years, all that we’ve done in 2012, and all that we’re doing today, my heart fills with…Continue
Those engaged in federal advocacy probably thought the Prescription Drug User Fee Act and its acronym PDUFA would take the cake…
Today, Parent Advocate and Founder of Two Smiles One Hope Foundation, Alison Willis and I represented the Duchenne community at the Congressional Rare Disease Caucus Briefing in Washington, DC. Alison did a remarkable job speaking on behalf of PPMD regarding the Patient Participation in Medical Product Discussions Provision within the newly passed…Continue
Added by Ryan Fischer on November 14, 2012 at 3:00pm — No Comments
Ivy's son, Rain
Last year, Ivy and I agreed to write a co-blog. I would introduce Ivy’s or wrap something…Continue
While many of us – particularly those of you who live in key "swing" states that will likely determine the outcome of the election – may be tired of the unceasing television commercials, robocalls and mailers, it's important to recognize that regardless of who is elected, creating…Continue
Added by Ryan Fischer on November 5, 2012 at 2:30pm — No Comments
Workplace giving campaigns are convenient and effective ways to raise funds in support of ending Duchenne. And they are underway now!
Let your family, friends, coworkers, and everyone on your email list know that the annual United Way and…
Added by Will Nolan on October 25, 2012 at 9:00am — No Comments
New release from Prosensa:
£10m in milestone payments received from GlaxoSmithKline
Leiden, The Netherlands, 23 October 2012 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, has selected clinical candidates for two more compounds for the treatment of Duchenne muscular dystrophy (DMD) and has been granted…Continue
I am very hopeful for better understanding, astrologically, another heavenly sign, far more important than Mars to study the muscles.
I thank the members of this community, having left Shashi…Continue
Added by Antônio Carlos Costa Lima Vieira on October 22, 2012 at 10:47am — No Comments