A randomized single and multiple ascending dose study in healthy volunteers of LTI-291, a centrally penetrant glucocerebrosidase activator.
Jonas M den HeijerAnnelieke C KruithofGuido van AmerongenMarieke L de KamEva ThijssenHendrika W GrievinkMatthijs MoerlandMike WalkerKees BeenRenato SkerljCraig JustmanLindsay DudgeonPeter LansburyValerie C CullenDana C HiltGeert Jan GroeneveldPublished in: British journal of clinical pharmacology (2021)
These first-in-human studies demonstrated that LTI-291 was well tolerated when given orally once daily for 14 consecutive days. This supports the continued clinical development and the exploration of LTI-291 effects in a GBA1-mutated Parkinson population.