Acid Sphingomyelinase Deficiency - Symptoms, Causes, Treatment
Por um escritor misterioso
Last updated 31 janeiro 2025
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
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