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.
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All in the FAMily
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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.