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.

Biomarkers
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
Biomarkers
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
Biomarkers
EAP
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
Biomarkers
Transcriptome of HPβCD-Treated Niemann-Pick Disease Type C1 Cells Highlights GPNMB as a Biomarker for Therapeutics

December 2021

Human Molecular Genetics

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If you have additional questions related to information provided on this website or other medical inquiries, please contact the Beren Medical Affairs team at medinfo@berentx.com.

The information provided on this website is provided by the Beren Therapeutics P.B.C. (“Beren”) Medical Affairs team for healthcare professionals in the US. It is designed to provide self-service access to medical information and medical affairs content about Beren products, pipeline, and therapeutic areas of interest including clinical data, research developments, congress materials, and publications. Certain materials such as posters and presentations presented in this website are a result of Beren-sponsored research and are not intended to be a comprehensive overview of all information and materials presented at a particular congress. The website is intended to provide scientifically balanced information and educational resources and may include information related to products or indications that have not been approved by the US Food and Drug Administration. The information provided is not intended as medical advice and does not replace independent medical judgement. By searching this website, you confirm the unsolicited nature of your inquiry. Please confirm that you agree and that you are a registered healthcare professional registered to practice in the US.

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