Viewing profile — fullburley

1. Give yourself some understanding and grace. You are a good parent, doing your best, never forget that. "How to Keep House While Drowning" I have found to be a good read to help …

For a marketing video that contains both untreated, and before and after DMD with this med, the outcomes are remarkable. https://youtube.com/watch?v=IolcG-a2yeY&feature=share8

The approved label reduced access to 4 and 5 year olds, shrinking the market opportunity in the short term.

Those were for the Muscular Dystophy Association, who cover DMD along with many other neuromuscular diseases.

As a first of kind treatment, they really didn't pick the right endpoints. Seeing videos of kids able to walk up stairs with confidence, after treatment, when they couldn't before,…

It is very expensive. We will likely have $2M USD in insurance claims for medical this year, and our son is currently aged out of the approved label. At a gross cost of $3.2M for t…

Serum CK testing would be a reasonable cost available surrogate test, if in normal range than muscle isn't being destroyed as one would see in traditional DMD cases. If high, run t…

In the case of DMD, it's a matter of when and which (typically heart), not if. The muscle fibers turn into fiberous tissues that aren't functional muscle. They fail.

That's for one type of muscular dystrophy, SMA, there are many other types without great treatment options. Lifelong exon skipping treatments recently approved for subsets of DMD a…