Few pieces of legislation may be as peculiar – and as important to the development of drugs and devices in this country – as the package of bills known as Food and Drug Administration (FDA) Prescription Drug User Fees Acts, known as PDUFA, or user fees for short. Born out of a frustration – amplified by the lack of therapies to respond to the AIDS epidemic – that FDA was taking too long to review drug applications, Congress enacted the first such law 20 years ago, in 1992. The law set out…Continue
Added by Ryan Fischer on May 17, 2012 at 10:28am — No Comments
Register for the 2012 Annual Connect ConferenceContinue
Added by PPMD on May 17, 2012 at 9:30am — No Comments
Cory Stalling is a fifth grader from Missoula, Montana. On July 8, a Run For Our Sons team will again pound the streets of Missoula on behalf of Cory as part of the Missoula Marathon. Cory and his family have been a big part of this community and now are taking another step to raise awareness, with a just…Continue
Getting it all done and figuring out what needs to be done first. Everyone, whether you are dealing with Duchenne or not, feels the pressure of time and faces a daunting, ever-growing to-do list. But when you’re dealing with a disorder like Duchenne, that clock ticks louder. In their monthly co-blog, PPMD President Pat Furlong and Colorado FACES coordinator, Ivy Scherbarth try to sort through priorities and what in our lives deserves more of our time and energy. Please take a moment…Continue
Thank you for participating in last week’s webinar on DuchenneConnect data! Many of you asked questions during the webinar that we were unfortunately unable to get to within the hour. We have done our best to answer all questions submitted.
Please see the list of Q&A's following the webinar video below, and let me know if you have any additional follow up questions!…
Added by Ann Martin on May 2, 2012 at 12:00pm — No Comments
One thing about conquering the canyon: before you see it, it seems ok, reasonable, even possible. The moment you see THE GRAND CANYON, you think WTF and how does this 'conquer' business really work and where was my head when I was laughing and taking bribes? On day one, we meet the guides (Gene and Jo Ann Taylor), who discuss various trails, share their experiences and answer…Continue
The Senate and House are moving closer to taking action on critical legislation focused on the Food and Drug Administration called PDUFA. Every five years Congress must pass a package of policies, known as user fees, that dictate the terms for the pharmaceutical and biotechnology and related industries to help fund…Continue
Added by Ryan Fischer on April 24, 2012 at 10:30am — No Comments
How many times have you explained something that happened, but in the retelling, the moment lost its punch?
I’m a fan of technology. Really. I love it that we are able to participate in meetings and conferences in real time, from anywhere in the world. It encourages, incentivizes,…Continue
Sometimes the best way to tell your story is through pictures. We can feel what another feels by seeing things the way they see them. Francis Desmond is an 8th grader who likes to draw. He also has Duchenne muscular dystrophy. Francis and his dad, Steven Desmond, decided to turn their journey into art and have just their father/son illustrated project, A…Continue
Added by Will Nolan on April 5, 2012 at 2:00pm — No Comments
Everyone is in a race against the clock these days. Nowhere is that more the case though, than in this community. Dealing with Duchenne, on top of everything else life throws at you, can leave you feeling overwhelmed, inadequate, and exhausted. In their monthly co-blog, PPMD President Pat Furlong and Colorado FACES coordinator, Ivy Scherbarth discuss the ways we cope with this ticking clock while keeping our families happy, our son’s healthy, and our sanity in check. Read this month’s…Continue
Are you trying to come up with an idea on how to fundraise for PPMD? Or has someone told you that they want to fundraise for PPMD, but they don't know what to do? Here are some really cool fundraising ideas that people are putting together right now in the community, which we're really excited about… Hopefully they inspire you!
Added by Danielle Garrigan on April 4, 2012 at 1:30pm — No Comments
This morning, AVI BioPharma released information discussing their findings thus far in their Phase IIb study of Eteplirsen.
Eteplirsen uses AVI’s phosphorodiamidate morpholino oligomer (PMO)-based chemistry to skip exon 51 of the dystrophin…Continue
Added by Ryan Fischer on March 30, 2012 at 9:30am — No Comments
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…Continue
Added by Pat Furlong on March 28, 2012 at 11:00am — No Comments
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…
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…Continue
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…Continue
Added by PPMD on March 19, 2012 at 2:30pm — No Comments
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.
Added by Pat Furlong on March 14, 2012 at 4:30pm — No Comments
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:…Continue
Added by Will Nolan on March 13, 2012 at 4:30pm — No Comments
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…
Added by Pat Furlong on March 13, 2012 at 10:30am — No Comments