Annemieke Aartsma-Rus has provided the following explanation to the recently published article in The FASEB Journal:

"DMD transcript imbalance determines dystrophin levels"

Explanation:

Since exon skipping modulates the amounts of dystrophin RNA transcripts (i.e. the DNA copies that are translated into protein), we…

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…

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.

A recent…

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…

“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 bottom number, or diastolic pressure).…

This summer, just prior to the 2013 Annual Connect Conference in Baltimore, a group of 40 parents, advocates, and professionals gathered for two days to discuss the needs, and how best to meet the needs, of individuals living with Duchenne.  Historically, Duchenne has been a pediatric diagnosis with no need to discuss adolescent and adult issues – thankfully, those…

my son has deletion of exon 45-47 , which exon skipping will be eligible for .. does deletion of 45-47 cause DMD or BMD ..

Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD and mom to Hazel, age 8, and Rain, age 6. Rain has Duchenne muscular dystrophy. Follow Ivy at her blog, Living …