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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...

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Principais autores: Mervat A. M. Youssef, Esraa Hefzy Shaker, Nahed A M Saleh
פורמט: Artigo
שפה:Inglês
יצא לאור: SpringerOpen 2025-08-01
סדרה:Molecular and Cellular Pediatrics
נושאים:
ASMD
Pediatric
Thrombocytopenia
Anemia
Enzyme replacement therapy
Olipudase alfa
גישה מקוונת:https://doi.org/10.1186/s40348-025-00199-9
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