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A mutation in the GBA1 gene is the most common genetic risk factor for developing Parkinson's disease. GBA1 encodes the lysosomal enzyme glucosylceramidase beta (glucocerebrosidase, GCase) and mutations decrease enzyme… Click to show full abstract
A mutation in the GBA1 gene is the most common genetic risk factor for developing Parkinson's disease. GBA1 encodes the lysosomal enzyme glucosylceramidase beta (glucocerebrosidase, GCase) and mutations decrease enzyme activity. LTI‐291 is an allosteric modulator of GCase, enhancing its activity. These first‐in‐human studies evaluated the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple ascending doses of LTI‐291 in healthy volunteers.
Keywords:
randomized single;
glucocerebrosidase;
single multiple;
lti 291;
healthy volunteers;
multiple ascending
Journal Title: British Journal of Clinical Pharmacology
Year Published: 2021
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Journal Title: British Journal of Clinical Pharmacology
Year Published: 2021
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!