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, …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
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…Continue
Added by Danielle Garrigan on March 12, 2012 at 4:30pm — No Comments
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.
Weakness. A familiar word in the context of…Continue
By now we hope you have heard that Parent Project Muscular Dystrophy will be hosting our first West Coast Connect Meeting, April 13-15, 2012 in San Diego, CA at the…Continue
Added by PPMD on March 7, 2012 at 10:00am — No Comments
Everyone gives something to somebody for something. Whether through acts of kindness, services, or hard-earned money, everyone has the opportunity to make the world a better place. No energy required.
Literally from the comfort of our couch, we are able to reach across the world to support others in need. 250,000 boys/young men have Duchenne. They live everywhere,…Continue
Added by Pat Furlong on February 22, 2012 at 3:00pm — No Comments
As you know, ensuring appropriate federal funding for Duchenne research has been a cornerstone of PPMD’s advocacy agenda over the past decade. Our persistence – your persistent and collective voice - has paid off with over $250 million directed to Duchenne research.
Today we now find ourselves at a pivotal moment with multiple potential therapies for Duchenne in various stages of the development process. We must now, once again, look forward to what is next for Duchenne.…
Added by PPMD on February 21, 2012 at 10:00am — No Comments
I don’t know about you, but when I am in DC or receive government related information, I am always about 3 acronyms behind in the conversation. PDUFA – this is one acronym you want to put in your memory bank for 2012. PDUFA – Prescription Drug User Fee Act.
This legislation is all about FDA, about what biotech’s/pharmaceuticals have to pay for the process, about rare disorders and incentives to develop drugs for rare conditions, and about the process of…Continue
West Coast Connect Meeting
First, PPMD is excited to host our first West Coast Connect Meeting, April 13-15, 2012 in San Diego, CA. This will be a great opportunity to…
Added by PPMD on February 13, 2012 at 4:30pm — No Comments
PPMD continues to bring you the most up-to-date care resources, so that you and your family and everyone who helps care for your son, has the information they need at their fingertips. Here are some recent updates we wanted you to be aware of:
Added by Holly Peay on February 13, 2012 at 11:00am — No Comments
I am so excited to announce a new sporting fundraiser we have this year called Ice Duchenne!
PPMD has partnered with the Central Collegiate Hockey Association Coaches (CCHA) this weekend, February 10th and 11th. The coaching staffs of all 11 teams will band together to help raise awareness for Duchenne and wear pins with the…Continue
Added by Danielle Garrigan on February 9, 2012 at 11:30am — No Comments
Biochemist Gunter Scheuerbrandt has been following exon skipping for the international Duchenne community for years. Read the latest update from one of this community’s greatest allies.
Added by PPMD on February 8, 2012 at 11:30am — No Comments