While our Duchenne advocates (like 10 year old Anthony, pictured right) are out on the Hill today, urging their Members of Congress to preserve critical funding for Duchenne research and education programs, we need YOU to take a moment and advocate from home with us!



All it takes is a call or an email to make sure your voice is heard! Check out our Take…

Everyone gives something to somebody for something. Whether through acts of kindness, services, or hard-earned money, everyone has the opportunity to make the world a better place. No energy required. 

Literally from the comfort of our couch, we are able to reach across the world to support others in need. 250,000 boys/young men have Duchenne. They live everywhere,…

I don’t know about you, but when I am in DC or receive government related information, I am always about 3 acronyms behind in the conversation.  PDUFA – this is one acronym you want to put in your memory bank for 2012.  PDUFA – Prescription Drug User Fee Act.

 

This legislation is all about FDA, about what biotech’s/pharmaceuticals have to pay for the process, about rare disorders and incentives to develop drugs for rare conditions, and about the process of…

PPMD President Pat Furlong and Colorado FACES Coordinator Ivy Scherbarth co-blog about vulnerability and what you risk when you open your heart to people. One the one hand, we risk fraud – a subject Ivy has been dealing with intensely with her family, as of late. But at the same time, our vulnerability allows us to love and be loved. Pat and Ivy explore two perspectives on a similar idea.

 

Vulnerability

By …

PPMD welcomes Dr. Padraig Wright from GlaxoSmithKline as our guest for a webinar on February 22, 2012 at 1pm eastern.

 

• Topic: Exon Skipping
• When: Wednesday, February 22, 1pm eastern

GSK2402968 is an antisense oligonucleotide…

Guest blog by Kathi Kinnett RN/CNP

 

Currently there is no newborn screening for Duchenne in the United States. However, groundbreaking work from Jerry Mendell’s lab at The Ohio State University may have a hand in changing this.

 

Mendell’s recent pilot study was funded by the Centers for Disease Control (CDC) and…