Clinical-economic evaluation of a screening for Pompe disease in children in the local conditions

• প্রধান লেখক: A. S. Kolbin (লেখক), I. A. Vilum (লেখক), Yu. E. Balikina (লেখক), M. A. Proskurin (লেখক), O. I. Karpov (লেখক)
• বিন্যাস: গ্রন্থ
• প্রকাশিত: Izdatelstvo OKI, 2021-02-01T00:00:00Z.
• বিষয়গুলি: article
• অনলাইন ব্যবহার করুন: Connect to this object online.

Pompe disease with late onset (PDLO) can't be diagnosed in time due to common symptoms with several neuro-muscular diseases. Screening and diagnostic measures could lead to efficacy enzyme replaced therapy (ERT) with alglucosidase alfa with aim of severe complications prediction. Screening has...