MPS I – Doença de Hurler / Doença de Scheie

Associação Portuguesa de Doenças do Lisossoma - APL

MPS I – Doença de Hurler / Doença de Scheie

“An online survey on burden of illness among families with post-stem cell transplant mucopolysaccharidosis type I children in the United States” – LINK

“Newborn screening in mucopolysaccharidoses” – LINK

“Short stature as a presenting symptom of attenuated Mucopolysaccharidosis type I: case report and clinical insights” – LINK

“Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome” – LINK

“Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison” – LINK A CONSULTAR

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