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...
সংরক্ষণ করুন:
| প্রধান লেখক: | , , |
|---|---|
| বিন্যাস: | Artigo |
| ভাষা: | Inglês |
| প্রকাশিত: |
SpringerOpen
2025-08-01
|
| মালা: | Molecular and Cellular Pediatrics |
| বিষয়গুলি: | |
| অনলাইন ব্যবহার করুন: | https://doi.org/10.1186/s40348-025-00199-9 |
| ট্যাগগুলো: |
কোনো ট্যাগ নেই, প্রথমজন হিসাবে ট্যাগ করুন!
|