Litcius/Paper detail

FDA approves first two drugs for rare Niemann–Pick disease

Asher Mullard

2024Nature Reviews Drug Discovery11 citationsDOI

Topics & Concepts

MedicinePharmacologyDrug approvalOrphan drugRare diseaseNiemann–Pick diseaseDrugDiseaseVirologyInternal medicineBioinformaticsBiologyLysosomal Storage Disorders ResearchAutoimmune and Inflammatory Disorders ResearchBiochemical and Molecular Research