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In this Phase I/IIa clinical trial, investigators administered antisense huntingtin oligonucleotides intrathecally to patients with early Huntington’s disease (HD), and demonstrated safety, tolerability, and dose-dependent reduction in CSF mutant huntingtin. This approach now is being tested for clinical efficacy in HD in a Phase III clinical trial.
Financial Disclosure: Neurology Alert’s Editor in Chief Matthew Fink, MD; Peer Reviewer M. Flint Beal, MD; Editorial Group Manager Leslie Coplin; Editor Jason Schneider; and Accreditations Manager Amy M. Johnson, MSN, RN, CPN, report no financial relationships relevant to this field of study.