An unbiased fly-powered drug repurposing screen for FAM177A1 deficiency turned up the generic ALS drug riluzole as the top hit and inspired a family-initiated, family-led pioneer N-of-1 study.
All in the FAMily
An unbiased fly-powered drug repurposing screen for FAM177A1 deficiency turned up the generic ALS drug riluzole as the top hit and inspired a family-initiated, family-led pioneer N-of-1 study.