Publication | Open Access

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial

Neurodegenerative DiseasesNeurobiology Of DiseaseClinical TherapeuticNiemann-pick DiseasePharmacologyClinical TrialsDegenerative DiseasePharmacotherapyNeurologyNeuropathologyMedicineType C1