Olipudase alfa for acid sphingomyelinase deficiency: Evidence of efficacy from a compassionate use program.
Overview & Synopsis
Open access biomedical research article published in Mol Genet Metab Rep.
Indexed from repository: PubMed Central
(
pmc.ncbi.nlm.nih.gov)
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Document Information
| Document Title | Olipudase alfa for acid sphingomyelinase deficiency: Evidence of efficacy from a compassionate use program. |
|---|---|
| Author / Organization | Gragnaniello V, Tagi VM, Carubbi F, Barbato A, Sibilio M, Fecarotta S, Pappalardo MG, Motta I, Sechi A, Scarpa M, Pession A, Fiori L, SIMMESN Italian Enzyme Replacement Therapy Study Group |
| Subject & Discipline | Mol Genet Metab Rep |
| Categories | Medicine & Health |
| Publication Year | 2026 |
| License / Rights | Open Access / Creative Commons |
| Resource Verification | Listed PDF (Verification Pending) |
| Original Host Website |
PubMed Central
(pmc.ncbi.nlm.nih.gov)
|
| File Format | application/pdf (Portable Document Format) |
| Language | EN |
| Catalog ID | DOC-116825 |
| Original Source Link | Visit Source Page on pmc.ncbi.nlm.nih.gov » |