Nemours Children's Health System

Comprehensive characterization and translational implications of the GalnsR384C mouse model of Mucopolysaccharidosis IVA

Abstract Mucopolysaccharidosis IVA (MPS IVA) is a lysosomal storage disorder caused by a deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS), leading to progressive accumulation of keratan sulfate (KS) and chondroitin-6-sulfate (C6S) and resulting in systemic skeletal...

Delayed methotrexate toxicity after initial clearance in a critically ill patient with severe fluid overload

Methotrexate (MTX) toxicity is a life-threatening complication in oncology patients. We report a 19-year-old male with newly diagnosed Burkitt lymphoma who received high-dose methotrexate as part of induction chemotherapy and initially met protocol-defined serum clearance criteri...