Medical Affairs
Publications
External links to publications from the adrabetadex – historically known as 2-hydroxypropyl-β-cyclodextrin or VTS-270 – clinical development program and expanded access program.
Utility of 24(S)-Hydroxycholesterol as a Proximal Biomarker to Monitor Long-Term Intrathecal Adrabetadex Therapy in Individuals With Niemann-Pick Disease, Type C1
December 2025
Molecular Genetics and Metabolism
Convergent Validity of the Fine Motor, Speech, and Cognitive Domains of the 5-Domain Niemann-Pick Disease Type C Clinical Severity Scale
June 2025
Journal of Child Neurology
Elevated Cerebrospinal Fluid Total Tau in Niemann-Pick Disease Type C1: Correlation With Clinical Severity and Response to Therapeutic Interventions
March 2025
Journal of Inherited Metabolic Disease
Low Risk Profile of Long-Term Repeated Lumbar Puncture for Intrathecal Delivery of 2-Hydroxypropyl-Beta-Cyclodextrin in Patients With Niemann-Pick Type C
July 2023
Pediatric Neurology
Neurofilament Light Chain in Cerebrospinal Fluid as a Novel Biomarker in Evaluating Both Clinical Severity and Therapeutic Response in Niemann-Pick Disease Type C1
March 2023
Genetics in Medicine
Consistently High Agreement Between Independent Raters of Niemann-Pick Type C1 Clinical Severity Scale in Phase 2/3 Trial
February 2022
Pediatric Neurology
Transcriptome of HPβCD-Treated Niemann-Pick Disease Type C1 Cells Highlights GPNMB as a Biomarker for Therapeutics
December 2021
Human Molecular Genetics
A Phase 1/2 Open Label Nonrandomized Clinical Trial of Intravenous 2-Hydroxypropyl-β-Cyclodextrin for Acute Liver Disease in Infants With Niemann-Pick C1
September 2021
Molecular Genetics and Metabolism Reports
Anesthetic Management of Pediatric Patients With Niemann-Pick Disease Type C for Intrathecal 2-Hydroxypropyl-β-Cyclodextrin Injection
April 2020
Pediatric Anesthesia
Long-Term Neuropsychological Outcomes from an Open-Label Phase I/IIa Trial of 2-Hydroxypropyl-β-Cyclodextrins (VTS-270) in Niemann-Pick Disease, Type C1
June 2019
CNS Drugs
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