A hopeful therapy for Niemann-Pick C diseases - The Lancet
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Lysosomal storage disorders: from biology to the clinic with
Biomolecules, Free Full-Text
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
A hopeful therapy for Niemann-Pick C diseases - The Lancet
Therapeutic intervention for Niemann–Pick type C disease must
Niemann‐Pick type C disease: molecular mechanisms and potential
A Second Chance at Life”: Can Gene Therapies Beat Rare Disease
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
Identification of Niemann-Pick C1 disease biomarkers through
Targeting the central nervous system in lysosomal storage diseases
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por adulto (o preço varia de acordo com o tamanho do grupo)