fullburley

4 ms·

1.
by fullburley 3y ago
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 here. 2. Find an expert, hopefully near your regio
2.
by fullburley 3y ago
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
3.
by fullburley 3y ago
The approved label reduced access to 4 and 5 year olds, shrinking the market opportunity in the short term.
4.
by fullburley 3y ago
Those were for the Muscular Dystophy Association, who cover DMD along with many other neuromuscular diseases.
5.
by fullburley 3y ago
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, is rather compelling as evidence. Evidence will a
6.
by fullburley 3y ago
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 this treatment, its expensive but definitely cost effective r
7.
by fullburley 3y ago
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 the genetic test, free in the US and Canada via Decode D
8.
by fullburley 5y ago
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.
9.
by fullburley 5y ago
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 are >1M USD per year, and result in dystrophin expres