Ten Year Anniversary!
It's hard to believe we are now in our tenth year of advocating for those living with H-ABC/Tubb4a! We have made meaningful headway but the process is slow and complicated. Nevertheless, we continue to drive forward. Here is what we have been doing.
- supported initial roll out of Tubb4a natural history study
- initiated sponsored research agreement for AAV gene therapy still in discovery
- database access to family support services nationwide
- ULF Ambassador partnership supporting families living with h-abc and other Leukodystrophies nationwide
- comprehensive bi-lingual website
- global support for newly diagnosed patients
- pending roll out of drug repurposing study
- raised up to $100,000 annually
- strong advocacy partnership with Industry leaders and other Leukodystrophy advocates
- family conferences and webinars
- unrelenting promotion and raising awareness with newsletter and media outreach
- supported first family treated for N-1 treatment who, now in the second quarter of treatment using an ASO, is doing well!