A hopeful therapy for Niemann-Pick C diseases - The Lancet
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Marta, with Niemann Pick C, fights for a cure with gene therapy
Biomedicines, Free Full-Text
The complexity of a monogenic neurodegenerative disease: More than
Diagnostic approach in adult-onset neurometabolic diseases
Identification of Niemann-Pick C1 disease biomarkers through
Frontiers Emerging Concepts in Vector Development for Glial Gene
Identification of Niemann-Pick C1 disease biomarkers through
Full article: New therapies in the management of Niemann-Pick type
Niemann–Pick disease, type C - Wikipedia
Niemann‐Pick type C disease: molecular mechanisms and potential
Pharmaceuticals, Free Full-Text
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
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por adulto (o preço varia de acordo com o tamanho do grupo)