3 adults treated for 24 weeks showed a mean 17.7-point improvement in ICARS, with improvement across all ataxia domains and no serious adverse events.
Small, open-label study — but an encouraging signal.
doi.org/10.1002/jimd...
#JIMD #CDG
3 adults treated for 24 weeks showed a mean 17.7-point improvement in ICARS, with improvement across all ataxia domains and no serious adverse events.
Small, open-label study — but an encouraging signal.
doi.org/10.1002/jimd...
#JIMD #CDG