Towards Precision Therapies for Inherited Disorders of Neurodegeneration with Brain Iron Accumulation.
Robert V V SpaullAudrey K S SooPenelope HogarthSusan J HayflickManju A KurianPublished in: Tremor and other hyperkinetic movements (New York, N.Y.) (2021)
This article provides an up-to-date review of the current literature about Neurodegeneration with Brain Iron Accumulation (NBIA), with a focus on disease pathophysiology, current and previously trialed therapies, and future treatments in development, including consideration of potential genetic therapy approaches.