A hopeful therapy for Niemann-Pick C diseases - The Lancet
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eBioMedicine, June 2023, Volume 92
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Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
![A hopeful therapy for Niemann-Pick C diseases - The Lancet](https://europepmc.org/articles/PMC6176479/bin/nihms-1507972-f0004.jpg)
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
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Biomedicines, Free Full-Text
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Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological
![A hopeful therapy for Niemann-Pick C diseases - The Lancet](https://www.thelancet.com/cms/attachment/1ee4075e-7d41-4a7d-94fa-cbf877818449/gr1_lrg.jpg)
Neurological manifestations of lysosomal disorders and emerging
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Marta, with Niemann Pick C, fights for a cure with gene therapy
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Current Clinical Applications of In Vivo Gene Therapy with AAVs
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Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a
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The Lancet, 14 October 2017, Volume 390, Issue 10104, Pages
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Experimental treatment for Niemann-Pick disease type C1 appears
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