PPMD President Pat Furlong and Vice President of Research Sharon Hesterlee are attending MDA’s Scientific Conference this week in Washington, D.C. Most of what was presented are results that were previously announced. However, there were some interesting presentations, as well as, results worth repeating, that we wanted to share. Sharon and Pat will continue to blog from the conference:

From Pat Furlong

Interview with Thomas Voit

While the content…

Below is the latest update PPMD has received from Dr. Rohit Batta, Global Medical Affairs and Patient Relations Leader in GSK's Rare Disease Unit :

The purpose of this communication is provide an update to you, as you have requested, on GSK’s plans for providing the option of continued access to drisapersen for boys completing the US Ph IIb 48 week study (DMD114876)…

Guest post by Gail D. McVicker, Grandmother of Jake (10) and Ryan (6) - Pennsylvania

Duchenne: An Intergenerational Issue

Five years ago, I did not know the world of Duchenne muscular dystrophy existed. Before my young grandsons were officially diagnosed in 2008, my daughter told me that some genetic…

PPMD received the email update below from Hans Schikan at Prosensa:

Dear all,

Today, at the Cold Spring Harbor conference in the US, Dr John Kraus of GSK gave an excellent presentation on the outcome of the 117 (DEMAND II) clinical trial. The primary endpoint in this exploratory study was met and a statistically and clinically meaningful difference of 35 meters in the six-minute walk test between the continuous dosing regimen of 6mg/kg per week and placebo was…

Below is the latest update PPMD has received from Dr. Rohit Batta, Global Medical Affairs and Patient Relations Leader in GSK's Rare Disease Unit :

To keep you updated on the progress of the drisapersen (previously GSK2402968) clinical development program, as you have requested, I am writing to inform you that the results of the GSK Phase IIb study investigating drisapersen as…

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

Living Duchenne: Silence

One interesting thing about silence is listening to…