Dear Friends,

Due to the recent hurricane we are going to postpone tomorrow's MD STARnet webinar.  

We will be in touch regarding the new date. Sorry for the inconvenience. 

Best, 

Ryan Fischer

PPMD

EVENT DETAILS…

Background 

Friends, we have been actively pursuing a regulatory strategy and have had two meetings with the neurology division at the FDA. We are currently working on the 'to do' list for the community and are trying to understand barriers/obstacles to approval from the FDA side, as well as, from the sponsor side of the issue. We are working a smart, targeted call to action. We have learned through years…

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…

1997 may not seem so long ago, but at times it feels like half a lifetime to me. That was the year my family learned Matthew’s motor difficulties were something physical therapy could not correct and the first time we heard the word Duchenne. Everyone’s world changes after diagnosis of a devastating chronic illness and…

Dr. Jerry Mendell, Director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children's Hospital and principal investigator of the Phase IIb eteplirsen study, presented the full set of 48-week data from…

In the clinical trial world, there are some things that divide us these days- most specifically, what mutation type your son has. However, we are united in wanting better ways to look for the benefits of potential drugs; who isn’t hoping for a day when the muscle biopsy is a relic from the…

In light of PPMD’s Cardiac Webinar Series, our Director of Care, Kathi Kinnett answers questions/concerns about Ventricular Assist Devices (VAD).

 …

These are the words that best describe PPMD’s research plan.

1. We aim to put better drugs into development for clinical trials because we can’t afford the failures.
2. We apply funding strategically to increase the speed of testing new drugs.
3. We prioritize and fund the testing in Duchenne of drugs that are already approved for other purposes.
   
   
   
   

Everything we do goes through this filter to make sure that we are achieving one of these…

Following closely on the heels of the good news about Sarepta’s exon 51 skipping strategy, PPMD is pleased to report that…

With the recent news of Sarepta's promising 48-week data, many have asked how accelerated approval may play into the FDA review of eteplirsen. Let's…

The 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…

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. The focus was genes, rare genetic disorders. While most of my days are…