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Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-?-Cyclodextrin.
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Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-?-Cyclodextrin.
Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-?-Cyclodextrin. Pediatr Neurol. 2018 03; 80:24-34.
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PubMed
subject areas
Adolescent
Cyclodextrins
Female
Humans
Infusions, Spinal
Male
Niemann-Pick Disease, Type C
Spinal Puncture
authors with profiles
Anne Hoffmann
Lisa LaGorio
Joan A. O'Keefe
Elizabeth Berry-Kravis