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The Food and Drug Administration (FDA) has approved onasemnogene abeparvovac-xioi (Zolgensma; Avexis, Bannockburn, IL) for the treatment of all types of spinal muscular atrophy (SMA) in children under age 2 years. Approved indications for onasemnogene, an SMN gene-replacement therapy, include all types of SMA. Treatment of presymptomatic children diagnosed through genetic screening is also approved. Not all states include testing for SMA in newborn screening panels, however. Onasemnogene is given intravenously in a single 1-time infusion. In 2 clinical trials (STRIVE [NCT03306277] and...
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...
Mary Motwani, PhD; and Deena Kuruvilla, MD
Umer Najib, MD, FAHS; Jessica Frey, MD; and David B. Watson, MD, FAHS, FAAN
Rashmi B. Halker Singh, MD; Amaal J. Starling, MD; and Juliana VanderPluym, MD