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A prodrug formulation of levodopa-carbidopa delivered as an intestinal gel (LCIG) (Duopa; Abbvie, Chicago, IL) is approved by the Food and Drug Administration (FDA) for treatment of motor fluctuations and dyskinesia in advanced Parkinson’s disease (PD). Interim analyses of data from a multisite international prospective study (NCT02611713) confirm that treatment with LCIG decreases “OFF” time (mean decrease -4.1 hours from baseline over 12 months). Dyskinesia was also reduced, as measured by a -12.7-point decrease from baseline on Unified Dyskinesia Rating Scale...
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....
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...
Lauren Doyle Strauss, DO, FAHS; and Scott Otallah, MD
Rashmi B. Halker Singh, MD; Amaal J. Starling, MD; and Juliana VanderPluym, MD
Niushen Zhang, MD