Parliamentary Duchenne Rare Exon Meeting Report 26th March 2013
In Spring of this year, PPMD and other Duchenne stakeholders from around the world, convened a meeting with the UK's Shadow Minister of Health, Andrew Burnham, at Parliament. The purpose was focused on Antisense oligonucleotides (exon skipping) and developing an expedited pathway for all…Continue
Added by Pat Furlong on March 27, 2013 at 4:30pm — No Comments
Mientras observamos los días pasar y nos abrumamos por momentos intrascendentes, nuestras mentes se ocupan en observar estereotipos sin sentidos, tratando de imitar costumbres insanas e inapropiadas.... Otros, observan la vida como un privilegio, como un don, donde todos sus días son una bendición y un momento mas de esperanza, aun y a pesar de las muchas dificultades que enfrentan, su fe no se desvanece y parecen enormes ante los ojos Dios... Estos tiene la maravillosa potestad de ver lo…Continue
Added by Gerardo Javier reynoso on March 24, 2013 at 3:20pm — No Comments
I’m at the point where I can understand Stockholm Syndrome which is where a hostage or captive develops a bond with their captor. For many years I’ve been held in the grips by Duchenne muscular dystrophy and I’m not implying that I have any fondness for the disorder, the opposite is more accurate. More simply said, it is when you become accustomed to doing things a certain way or are adjusted to a particular ‘normal’ any change in your life or routine is…Continue
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.
Summit's Press Release:
Summit today outlines its future plans for the continued development of utrophin modulators for the treatment of DMD. Utrophin modulation is a disease modifying approach that has the potential to treat all genetic forms of DMD. Summit’s…
Added by PPMD on March 21, 2013 at 9:55am — No Comments
I'm not even sure really where to begin........
Our family has been so fortunate to be involved with such a "beautiful" group of people.
Wyatt was diagnosed with Duchenne Muscular Dystrophy in Jan 2009. Since then, things…Continue
Added by Will Nolan on March 15, 2013 at 11:00am — No Comments
Help Bring Run for Our Sons to your own backyard.
I hear it all the time, “I want to do a fundraiser but not sure what to do or where to start” – I ask “have you thought of doing a 5K or fun run in your community?” – The rest is Run for Our Sons history!
5Ks and fun runs are fast becoming one of PPMD’s…Continue
Added by Ryan Fischer on March 14, 2013 at 1:30pm — No Comments
Does anyone have advice about fundraising for individual needs i.e. house modification for wheelchair; bathroom, tracking, etc? We want people to be able to write off donation, and us not have to pay taxes on what is raised.Any suggestion on a non-profit organization that might allow us to raise money under their umbrella? Or some other avenue?
We were thrilled that 119 parents/guardians were willing and able to complete the entire treatment preferences survey. We are analyzing the data. We are doing the first part of the data analysis in-house, and the second part with our health economist collaborator. Our plan is to present this information to the FDA, to industry and clinical…Continue
Added by Holly Peay on March 13, 2013 at 10:30am — No Comments
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
PTC Therapeutics, Inc. (PTC) today announced the successful completion of a $60 million financing led by Brookside Capital Partners Fund, L.P.
"This substantial financing enables PTC to continue to pursue the regulatory approval and delivery of ataluren to patients," stated Michael Schmertzler, Chairman of the Board. "PTC's Board of Directors welcomes the breadth of support and expertise of our new investors."…
Added by PPMD on March 7, 2013 at 2:00pm — No Comments
Sarepta Therapeutics has provided updates on eteplirsen and their next steps with exon skipping in their latest release: Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2012…Continue
Added by PPMD on March 7, 2013 at 1:30pm — No Comments
I am so excited that in just 6 weeks, Parent Project Muscular Dystrophy will be hosting our second West Coast Connect Meeting in San Diego, CA, April 26-28, 2013. This meeting will be hosted at the San Diego Marriott Mission Valley.…Continue
Added by Kimberly Galberaith on March 6, 2013 at 4:00pm — No Comments
I wish we had a Tree of Hearts. Healthy new hearts would hang on the high branches and devices on the lower branches – low hanging fruit! Established guidelines about who, what, in what circumstances, and where, accompanied by a simple set of instructions for physicians all over the world to understand, adapt, and provide appropriate care for these technologies. A…Continue
Added by Pat Furlong on March 6, 2013 at 10:00am — No Comments
Updated 3/07/14: 109 total members- New additions in bold below
We continue to make progress on the Reauthorization of the MD-CARE Act. Thanks to you, we have…
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
A person looking in
would see a happy little man.
A person walking by
would see this as a lie.
A person living here
would notice the tears.
Tears that fall
when he cant catch a ball.
Tears that soak his cheeks
When someone makes a joke.
Tears that only a mom can wipe away
And make things seem okay.
He's just a little boy
that need to enjoy his life.
He feels like everyone else does
Added by danielle on March 5, 2013 at 10:31am — No Comments