Ann: Appendix 4C & Quarterly Update, page-37

Hi all,

Does MPL work on every variant (100) . My opinion is no. Does it work on Bulbar variant , my thoughts are no. Does it work on the main variants which encompasses 70% my findings are yes.

Ok why doesn't it work on Bulbar. This has been known for some time.

Bulbar MND ALS is more insidious and quicker onset due to the cytoplasm in the ARpolyQ protein that may block the autophagic flux due to the misfolded protein overload in the cells.


https://www.frontiersin.org/journals/endocrinology/articles/10.3389/fendo.2019.00569/full#B51

https://www.sciencedirect.com/science/article/abs/pii/S019745800600176X

I had contact with a Bulbar patient that tried to get on the MEND study and was told by one of the Guru's that it wouldn't work and she wasn't eligible. Lady passed away 10 weeks ago.

Bulbar treatment is highly complex and if I had an any idea on treatment I would post it.

Kpax