Found: 3
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Twice weekly dosing with Sebelipase alfa (Kanuma®) rescues severely ill infants with Wolman disease.
- Published in:
- Orphanet Journal of Rare Diseases, 2024, v. 19, n. 1, p. 1, doi. 10.1186/s13023-024-03219-5
- By:
- Publication type:
- Article
The lysosomal storage disorders mucolipidosis type II, type III alpha/beta, and type III gamma: Update on GNPTAB and GNPTG mutations.
- Published in:
- Human Mutation, 2019, v. 40, n. 7, p. 842, doi. 10.1002/humu.23748
- By:
- Publication type:
- Article
Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease.
- Published in:
- 2021
- By:
- Publication type:
- journal article