Posted by PPMD on February 25, 2015 at 11:00am 0 Comments 1 Favorite
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Parent Project Muscular Dystrophy via Facebook
The recording of our Dystrophin 101 webinar is now available. Learn more about the basic biology of dystrophin, how dystrophin is measured in both preclinical studies and clinical trials, and the need for and role that dystrophin analysis play in clinical trials of novel agents designed to exert their therapeutic effects through an increase in dystrophin levels. Our speakers also discussed what is required in order to have dystrophin considered as a surrogate.…
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Carol Keskeny commented on Gisel Rivero's group Grief and Loss
Kimberly Lamon commented on Gisel Rivero's group Grief and Loss
Shelly commented on Annie Kennedy's blog post The Solid Suit: Combining Forces to Enhance Strength
David commented on Kathi Kinnett's blog post Important New Duchenne Steroid Protocol: Download the "PJ Nicholoff Steroid Protocol"
Kim S replied to Maria Salamon's discussion A Life in TransitionKai Mjaanes might attend PPMD's event
Parent Project Muscular Dystrophy via Facebook
Thank you to Olympic champion Ryan Lochte for supporting our fight to #EndDuchenne on #GiveRARE Day! Support the movement today and 100% of your donation will go to RESEARCH: http://giverare.razoo.com/story/Parentprojectmd
Parent Project Muscular Dystrophy via Facebook
Tuesday, March 3 at 1:00pm - 2:00pm EST: Our "Dystrophin 101" webinar will review the basic biology of dystrophin (the gene, structure-function of the protein, its role in the dystrophin-glycoprotein complex, characteristics of different forms of dystrophin (both naturally occurring and drug-induced truncated forms), discuss how dystrophin is measured in both preclinical studies and clinical trials, and discuss the need for and role that dystrophin analysis play in clinical trials…
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PTC Therapeutics today announced that it will make Translarna available for siblings of patients in certain open label PTC clinical trials for nonsense mutation Duchenne in advance of commercial availability in their regions. “Families of children with Duchenne have wanted companies to be responsive in the case of siblings. With this program, PTC, once again, demonstrates itself as a champion for the Duchenne community.” -- Pat Furlong, Founding President of PPMD…
See MoreParent Project Muscular Dystrophy via Facebook
PPMD is honored to join dozens of foundations participating in the first-ever Give RARE Day on March 3. Give RARE is a single day for the world to give to RARE disease. Although each disease is rare, in total 30 million Americans are affected by a RARE disease. None of us chose to be part of the RARE disease community, but here we are. And the Duchenne community is being looked at as a leader. Let’s show them what we can do as a community when we give. 100% of your donation will go to…
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