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.
Added by PPMD on October 30, 2012 at 1:00pm — No Comments
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 CFC efforts are underway (look for the 'share' button at…
Added by Will Nolan on October 25, 2012 at 9:00am — No Comments
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…Continue
What kind of fundraisers can we do to raise money for dmd??? Any help would be great...I would love to be involved and help find a cure!!!
Added by Laurie on October 23, 2012 at 3:18pm — No Comments
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…Continue
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…Continue
Added by Antônio Carlos Costa Lima Vieira on October 22, 2012 at 10:47am — No Comments
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 over the years my family and I experienced many…Continue
So sorry I haven't shared anything for some time. I have been very busy working on a new project that will promote called Halos for Muscular Dystrophy. I would like to welcome you each to join our online Facebook group: http://www.facebook.com/groups/HalosforMD/
HALOS FOR MUSCULAR DYSTROPHY. The Heart beyond the…Continue
Added by dreama whitlock on October 19, 2012 at 10:19am — No Comments
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…Continue
Added by PPMD on October 16, 2012 at 9:00am — No Comments
Wow! What an unbelievable few weeks we have had in this community! Never in the 18 year history of PPMD has the Duchenne community been so ripe with…Continue
Added by Pat Furlong on October 15, 2012 at 3:00pm — No Comments
Wheelz (a/k/a Ryan Schmidt) is a 20-year-old musician from Austin, Texas. He is an accomplished rapper, and with his family, hosts Ryan’s Day – one of the largest annual fundraising and awareness events for Duchenne in Austin, just held last week. The combination of his inspirational raps and Ryan’s Day remind people outside the community that we are in a fight to end…Continue
Added by Will Nolan on October 15, 2012 at 11:00am — No Comments
This month, instead of a pair of essays, Ivy Scherbarth and I are calling for a Virtual Moment of Silence. We invite you, our Duchenne/Becker Muscular Dystrophy community, to use the comments area here to list the names of the loved ones you remember and miss. We honor all…Continue
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 past?
There are several dedicated research teams doing exciting work…Continue
Added by Holly Peay on October 13, 2012 at 10:00am — No Comments
In light of PPMD’s Cardiac Webinar Series, our Director of Care, Kathi Kinnett answers questions/concerns about Ventricular Assist Devices (VAD).
Added by Kathi Kinnett on October 11, 2012 at 2:30pm — No Comments
These are the words that best describe PPMD’s research plan.
Everything we do goes through this filter to make sure that we are achieving one of these…Continue
Parent Project Muscular Dystrophy recently hosted the first part of our Cardiac Webinar Seriers with Dr. Linda Cripe (Nationwide Children’s Hospital, Columbus, OH) and Dr. Kan Hor (Cincinnati Children's Hosptial, Cincinnati, OH) to discuss monitoring heart disease in Duchenne and carrier moms/daughters.
Added by Kathi Kinnett on October 9, 2012 at 3:30pm — No Comments
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 first define what accelerated approval means.
When I was young, I remember my parents saying that “ignorance is bliss”. I never liked that expression and it has become more relevant for me when I speak with parents who have children affected by Duchenne muscular dystrophy who are trying to navigate educational or health care systems. Parents are often overwhelmed by the complexity of these programs and their…Continue
Added by Brian Denger on October 4, 2012 at 11:00am — No Comments
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 data about the dystrophin found in study participants’ muscle biopsies and the…Continue