A higher-dose nusinersen regimen for spinal muscular atrophy demonstrated statistically significant motor function improvement in treatment-naïve infants in the DEVOTE trial with a safety profile consistent with prior dosing.
Navepegritide is described as the first therapy to deliver continuous C-type natriuretic peptide exposure over a weekly dosing interval in eligible pediatric patients.
The US Food and Drug Administration approves pitolisant (WAKIX) tablets for the treatment of cataplexy in pediatric patients aged 6 years and older with narcolepsy.