First-ever approved treatment for Prader-Willi syndrome: Implications for pediatric nursing practice

Prader-Willi syndrome (PWS) is a rare, complex genetic disorder characterized by hypotonia, developmental delay, endocrine abnormalities, hyperphagia, and life-threatening obesity beginning in early childhood. Historically, management of PWS has relied on strict environmental control, nutritional supervision, growth hormone therapy, and behavioral interventions, with no disease-specific pharmacological treatment available. The recent approval of the first targeted therapy for Prader-Willi syndrome marks a major milestone in pediatric care and rare disease management. This advancement has significant implications for pediatric nursing practice, including treatment administration, growth and behavioral monitoring, family education, ethical advocacy, and multidisciplinary coordination. Pediatric nurses play a pivotal role in ensuring treatment adherence, early identification of adverse effects, and supporting family-centered care across developmental stages. This article reviews Prader-Willi syndrome in children, conventional management strategies, the newly approved treatment, and its nursing implications, highlighting the expanding role of pediatric nurses in improving outcomes and quality of life for affected children and their families.