Save the Date: Q&A Webinar with SareptaSarepta Therapeutics has asked PPMD to host a webinar on Tuesday, October 23 at 1pm eastern to discuss the 48-week results from their ongoing Phase IIb clinical trial of eteplirsen with the Duchenne community. This comes after early results were released yesterday. Promising News from Sarepta on Exon 51 Skipping TrialThe much-anticipated 48-week results on eteplirsen, a drug designed to skip exon 51, were presented today by Sarepta Therapeutics on a webinar and press release. On the webinar, Sarepta’s Chief Executive Chris Garabedian presented data about the dystrophin found in study participants’ muscle biopsies and the 6-minute walk results. PPMD President Pat Furlong receives RARE Champion Award in AdvocacyOn September 27, rare disease patients, advocates, patient organizations, government agencies, pharmaceutical companies, medical researchers, celebrities, Olympic champions and private sector representatives attended the Global Genes | R.A.R.E. Project 1st Annual "RARE Tribute to Champions of Hope" benefit. PPMD President Pat Furlong was honored to receive the RARE Champion Award in Advocacy. 
PPMD awards $50,000 to multi-exon skipping projectWe are so happy to support the work of Dr. Toshifumi Yokota at the University of Alberta, in Canada. Dr. Yokota is taking the exon skipping process several steps forward by experimenting with skipping multiple exons. If successful, a large percentage of the Duchenne population could be effectively treated. 2012 Coach To Cure MD PSAStarting tonight, coaches around the country over 10,200 to be precise! will wear a Coach To Cure MD patch to show their support of this community’s fight to end Duchenne. Over the next couple of days, let’s all do what we can to raise awareness and tell as many people as possible about our mission. Share this year’s Coach To Cure MD PSA through email, on Facebook and Twitter, or show it on your computer at work!September 27, 2012 | Watch the video 
Implementing PDUFAEarlier this year PPMD helped lead an effort supported by a number of other organizations to include within FDASIA key rare disease provisions intended to expedite review of potential treatments. But as all of you know, while enacting an idea into law is a major accomplishment, it's far from the end of the road. |
 
 Jeans. Genes. Thursday night, I changed from jeans into a blue dress as in blue jean blue. The recommended color for the R.A.R.E. Gala. October 1, 2012 | Read More  
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