Elizabeth Heller, is a woman living a full life – student, activist, wife – and does it all, living with Duchenne. Considered to be a disorder that predominantly affects males, Elizabeth is an exception. Each year, Elizabeth joins…Continue
It’s the official start of summer. And for our community, it’s that time of year when we come together with caregivers, scientists, and industry for PPMD’s Annual Connect Conference. This year’s agenda is quite astonishing, reflecting the vast number of therapies in the works, the updates to care, our increased presence in Congress, and a panel series that will dissect the clinical…Continue
Added by Pat Furlong on June 14, 2012 at 11:00pm — No Comments
We all feel like we are being held up to some predetermined standard – whether it is at our job, in school, or just walking down the street. In Duchenne, we compare our sons to other people’s sons, to others with Duchenne. But who is to say what is right or normal? In their monthly co-blog, PPMD President Pat Furlong and Colorado FACES coordinator, Ivy…Continue
Following recent overwhelming passage in the Senate, The Prescription Drug User Fee Act comes closer to becoming law after swift passage in the House of their version of the bill last night by a vote of 387-5. Both bills now move to conference for reconciliation.…
Added by Ryan Fischer on May 31, 2012 at 10:30pm — No Comments
As the U.S. Senate continues debate on legislation that seeks to accelerate delivery of safe and effective therapies to patients and to increase the patient voice as part of the therapy review process, PPMD is pleased to offer our enthusiastic endorsement of the legislation.
Over the past several months, PPMD has fought to include several key provisions included in the Senate version of the Food and Drug Administration Safety and Innovation Act, commonly referred to as the…Continue
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…