Did some DD on...

Did some DD on dystrophen


https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/206488lbl.pdf See section 14 of the eteplirsen Product Information leaflet. After 48 weeks of treatment eteplirsen only increased dystrophin levels from 0.16% of health patients to 0.44%. The median (mid-range) increase after 48 weeks was only 0.1%

Golodirsen increased mean dystrophin levels from 0.10% (SD 0.07) of normal at baseline to 1.02%(SD 1.03) of normal by Week 48 of Study 1 Part 2, with a mean change in dystrophin of 0.92% (SD 1.01) of normal levels (p<0.001); the median change from baseline was 0.88
https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/211970s002lbl.pdf

DMD patients (like the MDX mice) have very low levels of dystrophin protein and these Sarepta drugs increased dystrophin in piatients to about 1% of healthy subjects after 48 weeks of treatment - so very low levels to detect!

Imo: Some investors didn’t understand Mondays announcement…

Sam: once again it was a very good post can you elaborate)

Anp does not require Dystrophen to be successful.. But after the extended study if it does show a tiny distrophen increase in the mice : it will be important to others.eg financiers.
and Pharmas and we might see a spike in the sp..
Don’t forget Sarepta only produced micro Dystrophen not the real dystrophenp

imo
We wait.