A hopeful therapy for Niemann-Pick C diseases - The Lancet
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eBioMedicine, June 2023, Volume 92
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
Biomedicines, Free Full-Text
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
Neurological manifestations of lysosomal disorders and emerging
Marta, with Niemann Pick C, fights for a cure with gene therapy
Current Clinical Applications of In Vivo Gene Therapy with AAVs
Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a
The Lancet, 14 October 2017, Volume 390, Issue 10104, Pages
Experimental treatment for Niemann-Pick disease type C1 appears
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por adulto (o preço varia de acordo com o tamanho do grupo)