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.

EAP
NPCCSS
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
EAP
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
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
EAP
NPCCSS
Consistently High Agreement Between Independent Raters of Niemann-Pick Type C1 Clinical Severity Scale in Phase 2/3 Trial​

February 2022

Pediatric Neurology
EAP
Long-Term Treatment of Niemann-Pick Type C1 Disease with Intrathecal 2-Hydroxypropyl-β-Cyclodextrin

March 2018

Pediatric Neurology

Contact Beren Medical

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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