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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...
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...
Results from multiple trials of SMN1gene-replacement therapy (Zolgensma; Avexis, Bannockburn, IL) show promise for single-dose treatment of spinal muscular atrophy (SMA). In the STRIVE phase 3 study for treatment of spinal muscular atrophy, 22 infants under age 6 months, diagnosed genetically (biallelic SMN1 mutations/deletions, 2 SMN2 copies), were treated. Of the 15 babies treated more than 13.6 months or who discontinued the study before then, 13 (87%) survived without needing permanent ventilation, in contrast to the 25-50% survival rate for children with SMA when untreated....
Mary Motwani, PhD; and Deena Kuruvilla, MD
Charisse Litchman, MD, FAHS; and Sirisha Sanamandra, MBBS
Kate Onorato, MD; Carrie Dougherty, MD, FAHS; and Jessica Ailani, MD, FAHS