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
I'm sorry I have not been here for such a long time. So much has happened since we were here last.
Tim is doing well-better than expected. I can still him his roar of laughter from his room even with his door close. My son, Timothy, now 23 yrs old is so positive about life and full of laughter that he is not let letting this disease get to…Continue
Added by Dee on August 30, 2012 at 11:45am — No Comments
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.
Do any of your sons have GERD? My 17 year old was seen by the gastroenterologist in March..His complaint is that it feels like there is something stuck in his throat. She had him take prilosec, dyciclomine for upset stomach and polyethylene glycol powder to help with bowel movements..His stomach problems are stable now but he still feels the throat thing. We are going back to the doctor tomorrow but wondering if anybody else has dealt with this and how?
Added by Lisa Groeger on August 20, 2012 at 11:47am — No Comments
Added by Andrea Cleary on August 19, 2012 at 1:49pm — No Comments
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
I posted a new blog post. Please view it at:
Added by Christine Piacentino on August 13, 2012 at 8:21am — No Comments
As you read this month's newsletter, the 2012 Summer Olympics in London are coming to a close. The world’s best athletes are heading home with their medals or their hearts broken, but all of them taking with them memories that will last a lifetime. For this…Continue
Thank you to everyone who reached out to their Senate and House members about stopping consolidation of vital Duchenne programs at the CDC! Members of both the…Continue
In a letter to Senators Brown and Wicker (PDF), PPMD officially endorsed the National Pediatric…Continue
Added by Ryan Fischer on August 9, 2012 at 9:46pm — No Comments
The past six months have been action-packed for Run For Our Sons, and we wanted to take a moment to share what's happened and what's ahead with the entire Parent Project Muscular Dystrophy community. A big thank you to each and every one of you who have gone the distance to end Duchenne, whether it was a full marathon or a 5K - you're helping us raise funds to support PPMD's…
Added by Nicole Herring on August 9, 2012 at 8:46pm — 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
Creativity, humor, edgy, important – these are just some of the words we can use to describe our Featured Voice for August, Sean Quigg and his Spaghetti Arms project. Sean wanted to raise awareness in a clever way that explained Duchenne, but was also cool. He also wanted an excuse to follow some of his favorite bands. Thus, Spaghetti Arms was born, whose mission is to…Continue
Added by Will Nolan on August 9, 2012 at 7:26pm — No Comments
Over the past few years I have talked to a lot of parents, individuals with Duchenne, and clinicians about clinical trials. This probably won’t be a surprise to you: I often hear how desperation and a feeling of running out of time affect how people think and feel about clinical trials.
Many bioethicists are worried about…Continue
Added by Holly Peay on August 9, 2012 at 11:47am — No Comments
Parents agonize over clinical trials, balancing the pros and cons and above all else, wanting to do what is best for not just the physical health of their child, but the emotional health as well. In this month’s co-blog, Colorado FACES coordinator and parent Ivy Scherbarth and…Continue
Added by Pat Furlong on August 9, 2012 at 11:00am — No Comments
Rick Guidotti has a lot of energy - a lot of experience and expertise, but the first thing you notice about him is, he has a LOT of energy. I first heard him speak at Cincinnati Children's Hosptial Medical Center at pediatric grand rounds. He was in Cincinnati to promote his photography installation (“Positive Exposure”) at the Cincinnati Museum Center. He…Continue
Added by Kathi Kinnett on August 9, 2012 at 9:00am — 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