April 29, 2016
The Science is on our Side
A boy with Duchenne at Monday's FDA hearing on eteplirsen. On Monday, the FDA convened an advisory committee meeting to review eteplirsen, a precision medicine…
March 1, 2016
Is Eteplirsen a Miracle Drug?
For several years now, I have been advocating for the advancement and approval of a new drug called eteplirsen being developed by Sarepta Therapeutics. My…
February 5, 2016
A Fallen Solider
I feel crushed to have learned that the boy in the HT-100 trial who was recently hospitalized has died. We do not yet know the…
January 26, 2016
My Heart Aches
Although the fight against Duchenne is draining and difficult and often discouraging, I usually like to blog about the grit and determination we need to…
January 8, 2016
Massive Leap!
Today Akashi announced big news: a major partner has committed to fund the final steps to take HT-100 over the goal line to boys with…
December 24, 2015
Exon skipping up for Approval
Kyndrisa, the drug being reviewed today by an FDA advisory committee, is the first Duchenne treatment ever to go up for approval in the US.…
December 22, 2015
The Year of the Dragon: video blog
Today I share my first-ever "vlog", aka video blog. Don't worry, I had never heard the term either until someone much younger than me suggested…
November 23, 2015
Big Day Tomorrow
Tomorrow is a big day. A special advisory committee convened by the FDA will spend all day reviewing the first Duchenne treatment up for approval…
October 1, 2015
Inside Out
In the Pixar movie Inside Out, we get inside the brain of a girl named Riley and see her emotions come to life on the…