Platform trial design for neurofibromatosis type 1, NF2-related schwannomatosis and non-NF2-related schwannomatosis: A potential model for rare diseases

A. E. Dhaenens, Britt; Heimann, Günter; Bakker, Annette; Nievo, Marco; E. Ferner, Rosalie; Gareth Evans, D.; Wolkenstein, P.; Leubner, Jonas; Potratz, Cornelia; Carton, Charlotte; H. Iloeje, Uchenna; Kirk, George; O. Blakeley, Jaishri; R. Plotkin, Scott; J. Fisher, Michael; Kim, AeRang; Hernáiz Driever, Pablo; A. Azizi, Amedeo; C. Widemann, Brigitte; M. Gross, Andrea; Parke, Tom; Legius, Eric; Oostenbrink, Rianne

doi:10.1093/nop/npae001

Public

Platform trial design for neurofibromatosis type 1, NF2-related schwannomatosis and non-NF2-related schwannomatosis: A potential model for rare diseases

Published in Neuro-Oncology Practice • Jan 4, 2024

Authors:

Britt A. E. Dhaenens

Günter Heimann

Annette Bakker

Abstract

Background: -related schwannomatosis (grouped under the abbreviation "NF") are rare hereditary tumor predisposition syndromes. Due to the low prevalence, variability in the range, and severity of manifestations, as well as limited treatment options, these conditions require innovative trial designs ...

Subject

Neurofibromatosis type 2

Medicine

Neurofibromatosis

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