All Blog Posts Tagged 'duchenne' (62)

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.



Previously, Dr.…

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…

Parent Project Muscular Dystrophy, The Muscular Dystrophy…

Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD. Follow Ivy at her blog, Living Duchenne.…

Updated 4/11/13: From 55 to now 69 total cosponsors! New additions in bold below

We continue to make progress on the…

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…

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 …

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…

Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD. Follow Ivy at her blog, Living Duchenne.

Living Duchenne: My…

The 194^th 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…

Photo Credit

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I am often asked: "How can I help in the fight to end Duchenne?" and "What are things I can do to make a difference?" My answer is always…

Every Thanksgiving, I find myself with a lot to be thankful for: my family’s love and support, our Duchenne community, and your tireless efforts.

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…

PPMD Taking Action to Avert Sequestration

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…

Ivy's son, Rain

What IS Duchenne?

by Pat Furlong

Last year, Ivy and I agreed to write a co-blog. I would introduce Ivy’s or wrap something…

Tomorrow, we will head to the polls to choose who will lead our nation for the next four years.

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…

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…

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…

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…