2019/05/07 by Sarah J. Tabrizi, Blair R. Leavitt, G. Bernhard Landwehrmeyer +19 · 1 citation
Medicine · Neuroscience · #Adverse effect #Amyotrophic Lateral Sclerosis Research #Disease #Fibromyalgia and Chronic Fatigue Syndrome Research #Genetic Neurodegenerative Diseases #Huntingtin #Lysosomal storage disease #Mutant #Mutation
paper · pdf · doi:10.1056/nejmoa1900907
openalex publication_date 2019/05/07 · openalex created_date 2019/05/16 · openalex updated_date 2026/08/01
BACKGROUND: messenger RNA and thereby reduce concentrations of mutant huntingtin. METHODS: pharmacokinetics in cerebrospinal fluid (CSF). Prespecified exploratory end points included the concentration of mutant huntingtin in CSF. RESULTS: 10-mg, 30-mg, 60-mg, 90-mg, and 120-mg dose groups, respectively). CONCLUSIONS: to patients with early Huntington's disease was not accompanied by serious adverse events. We observed dose-dependent reductions in concentrations of mutant huntingtin. (Funded by Ionis Pharmaceuticals and F. Hoffmann-La Roche; ClinicalTrials.gov number, NCT02519036.).