Clinical and economic justification of screening for mucopolysaccharidosis type I in children at groups of risks
Background. Mucopolysaccharidosis Type I (MPS I) has clinical heterogeneity without specific symptoms leading to difficulties with diagnostic on time. In-depth screening for MPS I in children has aim of early detection and timely treatment with an enzyme replacement therapy. Aim. The purpose of this...
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Izdatelstvo OKI,
2021-12-01T00:00:00Z.
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