I have C9ALS, first signs March 2019, diagnosis March 2020. Having researched family tree I now note my mother, her sister and son, all had/have either ALS or FTD. I’m looking further back for indications.
My two sons are arranging genetic testing for themselves. My sister’s adult children now have to choose.
I am trying to get on the phase 1 trial of BIIB078/ CnRx in the hope that this will silence the multiple repeats of G4C2 in the C9orf72 gene.
So, I am very much in favour of genetic therapies to silence genes.
I am in the privileged position of having friends in senior positions in genetic therapy pharmas, so keep up to date with developments, I fear more so than my consultant.