Spinocerebellar Ataxia
Posted: Thu Dec 10, 2020 8:00 am
It seems a cure for Saki's condition might not be too far off.
I just saw a report on a successful treatment of an infant suffering from Spinocerebellar Ataxia Type 1
The treatment seems to be effective after only one injection - though that one injection is priced at a ridiculous 2,1 million $.
Luckily the child was born in Germany so the treatment was covered by health insurance.
https://www.frontiersin.org/articles/10 ... 80179/full
Type 1 is probably not the kind Saki suffers from (or she wouldn't have survived beyond infancy) but apparently similar treatments of other types are also in the works i.e. for Type 3 (which might be what Saki is suffering from).
http://www.uniqure.com/gene-therapy/pipeline.php
So if Saki manages to hold on till 2020 she might be okay after all.
I just saw a report on a successful treatment of an infant suffering from Spinocerebellar Ataxia Type 1
The treatment seems to be effective after only one injection - though that one injection is priced at a ridiculous 2,1 million $.
Luckily the child was born in Germany so the treatment was covered by health insurance.
https://www.frontiersin.org/articles/10 ... 80179/full
Type 1 is probably not the kind Saki suffers from (or she wouldn't have survived beyond infancy) but apparently similar treatments of other types are also in the works i.e. for Type 3 (which might be what Saki is suffering from).
http://www.uniqure.com/gene-therapy/pipeline.php
So if Saki manages to hold on till 2020 she might be okay after all.