Introduction
PWS, Prader–Willi syndrome, neurodevelopmental disorder, neurologic disorders
DYSFUNCTION, NEUROLOGIC DISORDERS
Prader–Willi syndrome (PWS) is a rare, complex, neurodevelopmental disorder due to lack of paternal chromosome 15q11.2-q13 expression [Dysfunction involving various hypothalamic systems may predispose patients with PWS to a number of symptoms [Due to the innate nature of PWS, patients usually develop growth hormone (GH) deficiency at infancy or during the childhood period, which led to the approval of recombinant human GH (rhGH) in PWS patients [In infants, toddlers, and young children (aged 4–38 months in published studies), rhGH therapy could improve motor strength, mobility, and body composition [Children have an enhanced capacity for brain plasticity compared with adults. The brain has high plasticity during its early development through pruning of the synapses and activity-dependent refinement of neuronal connections, and many pediatric neurologic disorders have an impact on the fundamental mechanisms of brain plasticity [To investigate the early use of rhGH on psychomotor development beyond its physical benefits, we conducted a phase 3 study to evaluate the efficacy in terms of motor and mental development, physical improvement, and safety of daily rhGH therapy in infants and young children with PWS in China.