THERAPEUTIC AREA
Niemann-Pick disease, type C (NPC)
Scientific and medical resources on Niemann-Pick disease, type C, a rare and fatal neurodegenerative genetic disorder caused by impaired intracellular cholesterol trafficking, and Beren’s research in infantile-onset NPC.
Publications & Science
Congress presentations and posters from Beren-sponsored research, along with links to external peer-reviewed publications on adrabetadex and NPC.
Efficacy of Intrathecal Adrabetadex in Infantile-Onset Niemann-Pick Disease Type C is Supported by Survival, Clinical, and Biomarker Data — Poster
A target trial emulation framework for externally controlled survival analyses ininfantile-onset Niemann-Pick disease type C — Poster
Early evidence of an emerging NPC treatment paradigm: adrabetadex use alongside arimoclomol and/or levacetylleucine — Poster
Medical Education
Disease state education for infantile-onset Niemann-Pick disease, type C (I-NPC) to support clinical understanding and informed decision-making.
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.
