When my teenage Charley was 3 years old, a nice doctor with a gentle smile handed him a death sentence. It was impossible to fathom,…

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…

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…

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…

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…

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…

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.…

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…

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…

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…