Lessons Learned From the Long-Term Use of Enzyme Replacement Therapy in the Treatment of Lysosomal Acid Lipase Deficiency
J Pediatr Gastroenterol Nutr
.
2022 Jun 1;74(6):726-727.
doi: 10.1097/MPG.0000000000003453.
Epub 2022 May 17.
Authors
Alanna Strong
1
2
,
Can Ficicioglu
1
3
Affiliations
1
Division of Human Genetics, Children's Hospital of Philadelphia.
2
The Center for Applied Genomics, Children's Hospital of Philadelphia.
3
Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
PMID:
35576532
PMCID:
PMC9296543
DOI:
10.1097/MPG.0000000000003453
No abstract available
Publication types
Research Support, N.I.H., Extramural
Comment
MeSH terms
Enzyme Replacement Therapy*
Humans
Sterol Esterase / therapeutic use
Wolman Disease* / drug therapy
Substances
Sterol Esterase
Grants and funding
T32 GM008638/GM/NIGMS NIH HHS/United States