Diseases & Diagnoses
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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...
Impel Neuropharma (Seattle, WA) is developing new therapeutic formulations for delivery of therapeutic compounds to the upper nasal cavity with their precision olfactory delivery (POD) device. The POD uses a propellant to deliver drugs to the upper nasal cavity, a new route of administration, which can provide higher bioavailability and rapid uptake vs traditional nasal sprays The POD device is easy to use, requiring only that the nasal tip of the device is inserted into the nostril and squeezed once to activate dose delivery. Coordination of breathing with activation is not required, and...
In a phase 2b/3 clinical trial (NCT02848326), adults treated with atogepant (Allergan, Madison, NJ) had greater reduction from baseline in monthly migraine days (MMD) on average, compared with those treated with placebo. No serious adverse events related to treatment occurred. Of those treated with atogepant, the frequency of treatment-emergent adverse events ranged from 57% to 66% across the treatment arms vs 49.5% for those who took placebo. Elevated ALT or AST (>3x the upper limit of normal) occurred in 10 participants (1.8%), balanced across the 6 treatment arms, including...
Andrea P. Lee, MD; Giulietta M. Riboldi, MD; Ilya Kister, MD; Jonathan E. Howard, MD; and Ritesh A. Ramdhani, MD
Kate Onorato, MD; Carrie Dougherty, MD, FAHS; and Jessica Ailani, MD, FAHS
Ronald Devere, MD, FAAN