Diseases & Diagnoses
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The National Institute on Aging (NIA) has provided a 4-year grant for a phase 2 study of T3D-959 (T3D Therapeutics, Research Triangle Park, NC), a novel, metabolic-focused treatment for Alzheimer’s disease (AD). The PIONEER study, which will begin patient dosing in early 2020, is a double-blind placebo-controlled efficacy and safety study that will enroll up to 252 adults with mild-to-moderate AD. Participants in the study will receive 1 of 3 different oral doses of T3D-959 or placebo for 24 weeks. In a phase 2a study (NCT02560753), 34 participants with...
The Food and Drug Administration (FDA) has approved amifampridine (Ruzurgi; Jacobus Pharmaceutical Company, Princeton, NJ) for treating Lambert-Eaton myasthenic syndrome (LEMS) in patients age 6 to 17 years. This is the first FDA approval of a therapy specifically for pediatric patients with LEMS, a rare autoimmune neuromuscular junction disorder. Use of amifampridine in pediatric patients is supported by clinical studies in adults with LEMS, pharmacokinetic data in adult patients, pharmacokinetic modeling and simulation to identify the dosing regimen in pediatric...
Using a wearable technology (PKG) (Personal Kinetograph; Global Kinetics, Portsmouth, NH) cleared by the Food and Drug Administration (FDA), over 40,000 reports from individuals with Parkinson’s disease (PD) have been recorded and used by more than 200 Parkinson’s care centers. The PKG provides objective ambulatory measures of PD symptoms, including bradykinesia, dyskinesia, and tremor. Analysis of baseline data from the PKG showed that 54% of people reporting clinical measures with PKG had uncontrolled but likely treatable bradykinesia and 10% had uncontrolled, but likely...
Umer Najib, MD, FAHS; Jessica Frey, MD; and David B. Watson, MD, FAHS, FAAN
Andrea P. Lee, MD; Giulietta M. Riboldi, MD; Ilya Kister, MD; Jonathan E. Howard, MD; and Ritesh A. Ramdhani, MD
Michelle L. Dougherty, MD, FAAN