Outcome of enzyme replacement therapy for hematological and visceral manifestations in children with acid sphingomyelinase deficiency: a single center experience in upper Egypt
Abstract Background Thrombocytopenia is the most common hematologic manifestation of acid sphingomyelinase deficiency (ASMD). The introduction of enzyme replacement therapy (ERT) represents significant progress in the treatment landscape of this disorder. This study presents the largest pediatric ca...
محفوظ في:
| المؤلفون الرئيسيون: | , , |
|---|---|
| التنسيق: | Artigo |
| اللغة: | Inglês |
| منشور في: |
SpringerOpen
2025-08-01
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| سلاسل: | Molecular and Cellular Pediatrics |
| الموضوعات: | |
| الوصول للمادة أونلاين: | https://doi.org/10.1186/s40348-025-00199-9 |
| الوسوم: |
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