3 ms·
1. They're using a modified protein 1/4th the size of the regular muscle protein. 2. They only showed they can hit a surrogate endpoint, not a clinical benefit
by tehjoker 3y ago
1. They're using a modified protein 1/4th the size of the regular muscle protein.
2. They only showed they can hit a surrogate endpoint, not a clinical benefit yet.
It'll be interesting to see what happens. I hope these kids have better lives as a result of this treatment.
- fullburley 3y agoAs 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 also be hard to come by in normal quantities given the disease rarity.
- fullburley 3y agoFor 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 https://youtube.com/watch?v=IolcG-a2yeY&feature=share8