Propranolol, the current standard of care for essential tremor (ET), has significant shortcomings in efficacy and safety. As medicinal chemist Kevin Hodgetts, PhD, explains, Brigham investigators are developing novel therapies for ET patients by applying innovative drug design concepts to validated disease targets.

The Brigham’s AVM Program aims to provide the best possible nonsurgical and surgical treatment options for people with brain arteriovenous malformations (AVMs). Director Nirav J. Patel, MD, discusses his team’s use of an innovative surgical technique to treat AVM and efforts to set up similar programs internationally.

Researchers at Brigham and Women’s Hospital are investigating therapeutic focused ultrasound to enhance antibody delivery to the brain when treating Alzheimer’s disease.

The Brigham’s Fengfeng Bei, PhD, and team have reported on a new adeno-associated virus variant that delivers gene therapy across the blood-brain barrier in non-human primates more efficiently than previously developed vehicles. It could have implications for treating neurodegenerative diseases including Alzheimer’s.

Giovanna S. Manzano, MD, of the Department of Neurology at Brigham and Women’s Hospital, Marcelo Matiello, MD, of Massachusetts General Hospital, and colleagues determined positive predictive values of a live cell–based serum assay that detects the diagnostic biomarker for MOG-AD in a real-world cohort.

Brigham investigators have mapped out a group of cells in the trigeminal ganglia that drive migraine pain, allowing them to further characterize the cells in a way that holds promise for developing new therapeutics. Neurologist and lead author William R. Renthal, MD, PhD, elaborates on the study.

Vikram Khurana, MD, PhD, and colleagues have discovered an additional function of α-Synuclein—it modulates the stability of mRNA, thereby regulating gene expression. They explain the implications for understanding normal cellular physiology and developing therapies for Parkinson’s disease and related disorders.