Olipudase alfa treatment for pediatric acid sphingomyelinase deficiency in Egypt: A prospective, observational cohort study with an interventional subgroup

This case series contributes to the clinical and biochemical understanding of ASMD and may help reduce diagnostic delays and improve clinical management. Olipudase alfa was generally well tolerated, and promising clinical improvements were observed.

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Publication Details

Journal
Molecular Genetics and Metabolism Reports
Published
2026-02-11
DOI
https://doi.org/10.1016/j.ymgmr.2026.101293
Primary Topic
Sphingolipid Metabolism and Signaling
Type
article
Field-Weighted Citation Impact
0.00
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article

Olipudase alfa treatment for pediatric acid sphingomyelinase deficiency in Egypt: A prospective, observational cohort study with an interventional subgroup

Nehal Abdelaziz Arafa, Iman Marzouk, Shimaa Anwar, Aml Mahfouz
Molecular Genetics and Metabolism Reports
Sphingolipid Metabolism and Signaling
article

Olipudase alfa treatment for pediatric acid sphingomyelinase deficiency in Egypt: A prospective, observational cohort study with an interventional subgroup

Nehal Abdelaziz Arafa, Iman Marzouk, Shimaa Anwar, Aml Mahfouz
article en

Abstract

This case series contributes to the clinical and biochemical understanding of ASMD and may help reduce diagnostic delays and improve clinical management. Olipudase alfa was generally well tolerated, and promising clinical improvements were observed.

Molecular Genetics and Metabolism ReportsVol. 46
Alexandria University (EG)
Openalex Percentile: Top 59%
Sphingolipid Metabolism and Signaling
DOIOpen Access PDFOpenAlex
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Olipudase alfa treatment for pediatric acid sphingomyelinase deficiency in Egypt: A prospective, observational cohort study with an interventional subgroup — Nehal Abdelaziz Arafa, Iman Marzouk, et al. · Molecular Genetics and Metabolism Reports (2026) | TGRS Research Map | TGRS