Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1

Use of 2 hydroxypropyl-beta-cyclodextrin therapy in two adult Niemann Pick Type C patients

(2016) Ana Alejandra García-Robles et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

Phase 1/2 evaluation of intrathecal 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick disease type C1

(2016) Forbes D. Porter et al.   MOLECULAR GENETICS AND METABOLISM

Cyclodextrins as Emerging Therapeutic Tools in the Treatment of Cholesterol-Associated Vascular and Neurodegenerative Diseases

(2016) Caroline Coisne et al.   MOLECULES

Cholesterol Depletion from a Ceramide/Cholesterol Mixed Monolayer: A Brewster Angle Microscope Study

(2016) Pritam Mandal et al.   Scientific Reports

Diagnostic workup and management of patients with suspected Niemann-Pick type C disease

(2016) Apostolos Papandreou et al.   Therapeutic Advances in Neurological Disorders

Efficacy and ototoxicity of different cyclodextrins in Niemann-Pick C disease

(2016) Cristin D. Davidson et al.   Annals of Clinical and Translational Neurology

Broadscale resolving power performance of a high precision uniform field ion mobility-mass spectrometer

(2015) Jody C. May et al.   ANALYST

Complex lipid trafficking in Niemann-Pick disease type C

(2014) Marie T. Vanier   JOURNAL OF INHERITED METABOLIC DISEASE

Niemann-Pick disease type C symptomatology: an expert-based clinical description

(2013) Eugen Mengel et al.   Orphanet Journal of Rare Diseases

Weekly Cyclodextrin Administration Normalizes Cholesterol Metabolism in Nearly Every Organ of the Niemann-Pick Type C1 Mouse and Markedly Prolongs Life

(2010) Charina M Ramirez et al.   PEDIATRIC RESEARCH

Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression

(2009) Cristin D. Davidson et al.   PLoS One