We are coming to you today to ask that you devote your attention and energy towards legislation that seeks to expedite the process FDA uses to review drug applications.…

Bikram yoga. Michelle had just been home talking about her Bikram practice. We talked about it a lot over the holidays. Michelle had been practicing for nearly 5 years. I wondered if I could survive 90 minutes in 105 degree heat. I had a range of readily available excuses; the easiest one was that there was no Bikram in Cincinnati, Ohio. And my unspoken reason was that I hesitated because I…

 Last December when I was training for the 2012 Goofy run at WDW I participated in a 25k trail race. This was supposed to be the last big training event and then I would begin tapering for WDW.

 I love trail running, I hit the trails every chance I get and this race was taking place right where I run every week so I was excited to really do well. We all lined up and away we went looping around a small lake before we hit the trails. Ah the trails, my home away from home. This is where…

Andrew just started repeating this out of nowhere during dinner the other day.

 In case that didn't work, here's the link youtube

As always, you can follow along with us at www.HomemadeTaterTot.blogspot.com

In parallel to the on-going development of the lead compound PRO051/GSK2402968, which is being developed by GSK with an active involvement of Prosensa, there are five more compounds for Duchenne in Prosensa’s pipeline.  Below you can find an update about the current state of development of these compounds.

Prosensa is currently conducting a clinical study to investigate the effect of PRO044 in boys with Duchenne muscular dystrophy, who have a dystrophin gene mutation amenable to…

Josiah sat next to me brushing his teeth. While  I brushed my hair looking in  his large bathroom mirror,  I glanced over at him.  I was  so happy and proud that  he could still manage this task by himself. Reaching over towards him I placed a free standing counter mirror in front of him, just in case he wanted to see for himself the beautiful job he was doing. Josiah kept busy with his grooming and never glanced over at the mirror.   Smiling at him I suggested he see how gleaming white his…

I’d like to say this has “been one of those days”, but if the truth be known it’s more like one of those years.  Life for families affected by Duchenne muscular dystrophy, I have to guess like life for most other people, has its share of ups and downs.  The difference for those living with Duchenne is that it seems once you begin to feel some stability in your life the proverbial rug gets pulled from beneath your feet and reality not only…

Steroids. What regimen, what dose, for what individual? When to start? When to stop? A complicated question to be sure.

 

Steroids are a complicated story and have clearly changed the natural history of Duchenne. We know they work for some, for most in fact, but not all. We know they increase strength and prolong ambulation, and statistics back this up. Predicting ambulation is prolonged on average for 2+ years. But this is not the end of the story. Genetic modifiers have been…

AVI Clinical Trial Update

Parent Project Muscular Dystrophy presents an AVI webinar on Wednesday, March 21 at 1pm eastern. The webinar will cover a pre-data update on AVI BioPharma's Phase IIb study evaluating eteplirsen for the treatment of Duchenne patients with mutations amenable to exon-51 skipping, and will be lead by Chris Garabedian, the CEO of AVI…

Jon’s second semester at college has been rewarding and frustrating.  While Jon is on campus, things are a little easier.  Jon has a power chair that is stored on campus and he can navigate independently.  Jon doesn’t have a power chair yet permanently.   He can still walk and get in and out of a regular car, with a little help.  However, getting power chair of his own and an…

So many times we view the world from the dark side, the part where we worry our sons (and daughters) may never see x or y, may never have this or that opportunity. I recall Kevin Smith standing up and telling us how he promised Zach there would be no limits on what he wanted to do or see, that if he wanted to see the top of a mountain, Kevin would put him on his back and show him the view. I think any of us would do that for our child or for our friend.

 …

Our Featured Voice for March 2012 is 30-year-old, Canadian author, Ricky Tsang. If you are part of the Facebook world, you probably are friends with Ricky or have at least seen his name around. He is the author of the recently released, RIDICULOUS: The Mindful Nonsense of Ricky’s Brain, which has been…

This is my first visit to San Francisco, the city by the bay, home of the Golden Gate Bridge and Tony Bennett’s song “I Left My Heart in San Francisco,” a song from my younger days and one that, even today, sometimes gets in my head and repeats and repeats.



Some time back I was invited to participate in the Rare Disease Leaders’ Forum, participating in a working…

Do you love to go out to eat? Do you want to help fundraise, but don't have the time? Then this is the event for you!! You can participate whenever you visit a new restaurant without doing extra work! It's fast and easy!! 

Dine Out for Duchenne is a week-long, online event where people can…

http://www.facebook.com/l.php?u=http%3A%2F%2Fbikestory4cory.blogspot.com%2F&h=TAQHTMDv6

An inspiration--two young men are going to cycle cross-country for their senior project. And at the same time they will raise funds and awareness of Duchenne. They plan to meet up with the Jett riders halfway through and finish their trip en masse.

PPMD President Pat Furlong and Colorado FACES Coordinator Ivy Scherbarth discuss the bravery of their sons, and all young men fighting Duchenne. Please, share your thoughts after reading this month’s co-blog.

Strength

by Pat Furlong

Weakness. A familiar word in the context of…