Acid Sphingomyelinase Deficiency - Symptoms, Causes, Treatment

Sphingomyelinase Deficiency: Practice Essentials, Pathophysiology, Etiology

AAV8-Mediated Hepatic Expression of Acid Sphingomyelinase Corrects the Metabolic Defect in the Visceral Organs of a Mouse Model of Niemann–Pick Disease: Molecular Therapy

The incidence of acid sphingomyelinase deficiency (ASMD) in cases of suspected Gaucher disease, genotype-phenotype correlation together with Lyso-SPM biomarker - ARCHIMEDlife

Niemann Pick Disease - Rivin

Sanofi Presents Research Advances in Rare Genetic Diseases at 15th Annual WORLDSymposium™ 2019

Sphingomyelinase Deficiency: Practice Essentials, Pathophysiology, Etiology

First treatment for Acid Sphingomyelinase Deficiency approved by US FDA

PDF) Impact and burden of acid sphingomyelinase deficiency from a patient and caregiver perspective

CHMP recommends Sanofi's Xenpozyme for rare disease ASMD - PharmaTimes