A Milestone in Rare Disease Treatment: FDA Approves First-Ever Drug for Prader-Willi Syndrome’s Hung

Akrutisinha03 Nov, 2025Health

Prader-Willi Syndrome (PWS) is a rare genetic disorder that can affect individuals of any age and gender. Generally, the symptoms appear in infancy or early childhood and are often diagnosed by the age of six. PWS impacts multiple systems, particularly the brain, hormones, and metabolism, which makes it complex to treat (Angulo et al., 2015). While future gene-editing tools like CRISPR offer long-term hope, current therapies remain limited due to the intricate genetic origins of the disorder. (Bohonowych et al., 2019)