Acid sphingomyelinase deficiency: Burden of disease and real world impact of enzyme replacement therapy on pediatric patients and caregivers.
- Resource Type
- Article
- Source
Molecular Genetics & Metabolism . Feb2023, Vol. 138 Issue 2, pN.PAG-N.PAG. 1p.- Subject
- *
ENZYME replacement therapy
*NIEMANN-Pick diseases
*DEFICIENCY diseases
*PEDIATRIC therapy
*CHILD patients - Language
- ISSN
- 1096-7192