Targeted treatment of hypophosphatemia with trametinib in HRAS-related mosaic RASopathy
Orphanet J Rare Dis. 2025 Jul 3;20(1):339. doi: 10.1186/s13023-025-03801-5. ABSTRACT Schimmelpenning-Feuerstein-Mims syndrome (SFMS) is a rare mosaic RASopathy associated with epidermal nevi, neurological abnormalities, and …