When parents of children with Duchenne enroll their children in a clinical trial there is often a sense of participating in something bigger than themselves—of course you hope that your child may benefit from a new therapeutic approach, but you also know that all those days of missed school,…Continue
Added by Sharon Hesterlee on October 29, 2013 at 11:00am — No Comments
When Chris and Patrick were diagnosed, there was no genetic testing. No therapies were targeted to the “CODE” and no way to DECODE Duchenne. We made assumptions about carrier status and lived many years with only assumptions. I thought my mother was a carrier. I knew I was a carrier. The doctor made that…Continue
Added by Pat Furlong on October 23, 2013 at 10:30am — No Comments
Added by Pat Furlong on October 17, 2013 at 9:00am — No Comments
When a family hosts a fundraising event for Duchenne, we are thrilled and extremely grateful. When a family hosts the same event six years in a row, we are overwhelmed! This year the Pritchard family, along with their friends – Meghan and Jason Alpern – in Matthews, North Carolina will host the…Continue
Added by Will Nolan on October 15, 2013 at 11:30am — No Comments
We all know it: rare diseases, especially progressive pediatric disorders like Duchenne, are different than common diseases. The FDA knows it too, but they don’t have a plan for how to treat them differently. They are working on different ways to weigh the benefits and risks of treatments for…Continue
Added by Holly Peay on October 10, 2013 at 9:30am — No Comments
I was privileged to share some of the data PPMD has collected in our Clinical Trial Expectations study at the World Muscle Society poster session. Many clinicians and industry and academic scientists came to the poster to learn about our community thoughts and preferences, and were quite interested in how they might…Continue
Added by Holly Peay on October 7, 2013 at 10:58am — No Comments
Several poster sessions, as well as the first portion of the Sarepta Symposium,…Continue
Added by Kathi Kinnett on October 4, 2013 at 12:59pm — No Comments
The World Muscle Society Meeting in Monterey, California saw reports on a number of topics of interest to the community. On Thursday poster sessions covered advances in the next generation of exon-skipping and updates on various clinical trials.
Next Generation Exon-Skipping…Continue
Added by Sharon Hesterlee on October 4, 2013 at 9:30am — No Comments
Prosensa has provided the following update regarding the impact of the drisapersen phase 3 data on its programs:
While we are disappointed and surprised with the unexpected outcome of the drisapersen phase 3 study, we remain very committed to drisapersen, the Duchenne program at…
Added by PPMD on September 24, 2013 at 11:30am — No Comments
UPDATE AS OF 10/4/13: We received the following Q&A from GSK today, addressing many of the community's questions. Click here.
Today we learned that GSK’s phase III study of the exon 51 skipping drug drisapersen failed to show a statistically significant improvement in the…Continue
Parent Project Muscular Dystrophy (PPMD) has been invited to present at two upcoming stakeholder meetings.
The invitations, to PPMD Founding President & CEO Pat Furlong, were extended by leaders in the U.S. government in recognition of PPMD's groundbreaking work to advance the patient and parent perspective in the drug development and review…
Added by PPMD on September 10, 2013 at 9:44am — No Comments
Guest post by Jill Anne Castle, mom to Oliver, 11, and Anthony, 13 and living with Duchenne. Jill is an Educational Consultant and PPMD FACES of Arizona Volunteer Coordinator.…
The Muscular Dystrophy Coordinating Committee (MDCC) had its 10th meeting on August 26, 2013 at the National Institutes of Health offices in Bethesda, MD. The members of the MDCC come from federal agencies involved in one or more muscular dystrophy programs as well as public representatives from several organizations representing one or more forms of muscular dystrophy. There are representatives from…Continue
Added by Brian Denger on September 4, 2013 at 3:00pm — No Comments
It’s that time of year again – the start of another school year. While we put our faith in schools to provide a proper, inclusive education for our children, faith is not all we have in our arsenal for ensuring this is the case for students living with Duchenne.
As parents, you are…Continue
Added by Ryan Fischer on September 3, 2013 at 10:45am — No Comments
Saturday, August 17 marks the 15th Annual Mitchell's Run Thru Rockford. And for 15 years, the Peterson family has been raising money and awareness for Duchenne. Mitchell Peterson, 17 years old, has been the inspiration behind this event that continues to grow in numbers and momentum. He refuses to be defined by Duchenne and as he begins his senior year of high school, is already planning for a career after college. The Peterson…Continue
Added by Will Nolan on August 15, 2013 at 12:29pm — No Comments
Obtaining physical therapy (PT) services for individuals with Duchenne and Becker muscular dystrophy, and other conditions for which PT does not result in improvements in range of motion or function, has been a huge source of frustration for patients, parents, and providers.
Added by Kathi Kinnett on August 15, 2013 at 8:00am — No Comments
PPMD announced today that it will fund up to $1 million in projects by mid-2014 focused on the development of antisense oligonucleotides or other techniques to skip exons that are not currently in clinical development, or for multi-exon skipping projects, including the skipping of…Continue
“Hypertension” is the fancy word for high blood pressure. Blood pressure is the measure of how much pressure it takes the left ventricle of the heart to squeeze the blood out (the top number, or systolic pressure) and the pressure inside the left ventricle of the heart when the heart is relaxed (the…Continue
As we move into the start of 2014 and the February Advocacy Conference we are hopeful to gain momentum to move MD-CARE Act to committee action this year. With 78 cosponsors in the House and 17 in the Senate, we need your help to up our…Continue
Added by Ryan Fischer on July 31, 2013 at 9:30am — No Comments