We are all acutely aware of the need for an effective treatment for Duchenne. Finding a way for children and young men affected by Duchenne to lead longer, healthier lives is at the heart of all of our efforts as families’ fight this devastating disease.
This week PPMD received an…Continue
One approach to improving muscle strength in Duchenne is to replace unhealthy muscle cells that lack dystrophin with donor stem cells that have normal levels of dystrophin. These cells might come from a relative unaffected by Duchenne or even a person with Duchenne’s own cell, after dystrophin is restored though a technique like gene therapy.
First, my disclaimer. I’ve known Scott and the Sands family for more than 20 years. I met Scott’s father during my travels long ago, both of us looking for magic for our sons. I knew well the story of Scott’s family traveling to Florida when he was 23, the emergency tracheostomy, at that time thought to be the best method for clearing secretions and battling…Continue
Added by Pat Furlong on August 15, 2012 at 8:00am — No Comments
Brian Denger is the father of two sons who are living with Duchenne. He is very involved with PPMD in the effort to educate and help families living with Duchenne.
Vacations and summer are synonymous for many families. When people think of vacations, what often comes to mind is travel to a tourist destination,…Continue
Added by Brian Denger on August 14, 2012 at 9:00pm — No Comments
Football season is one of my favorite times of year. It brings me back to watching my college football team, the James Madison University Dukes, who will always be near and true to my heart. Who doesn't like to tailgate and root on their teams?
On September 29, 2012, coaches from your favorite university will be wearing…Continue
Added by Danielle Garrigan on August 9, 2012 at 8:00pm — No Comments
As Sarepta (formely AVI) announces promising results from its phase II interim data and GSK follows up its promising phase…Continue
The clear message received by all at the end of the Transforming Duchenne Care meeting, held just prior to the PPMD Connect Conference, was that parents, providers and industry felt a need for clear, consistent standards of care for patients with Duchenne…Continue
Added by Kathi Kinnett on August 8, 2012 at 9:30am — No Comments
If you asked me if I could swim, I might say ‘more or less’. Translated, that means that somewhere long ago I took some lessons, that are now lost to memory and in some strange way in my ‘muscle memory’ and adapted for the few times I have access to a pool. For some of us, the ‘more or less’ approach is what we do. Paddle around the shallow end, jump or dive…Continue
Added by Pat Furlong on August 1, 2012 at 9:30am — No Comments