Cannabidiol (Epidyolex®) for severe behavioral manifestations in patients with tuberous sclerosis complex, mucopolysaccharidosis type III and fragile X syndrome: protocol for a series of randomized, placebo-controlled N-of-1 trials

Cannabidiol (Epidyolex®) for severe behavioral manifestations in patients with tuberous sclerosis complex, mucopolysaccharidosis type III and fragile X syndrome: protocol for a series of randomized, placebo-controlled N-of-1 trials

Abstract Background Many rare genetic neurodevelopmental disorders (RGNDs) are characterized by intellectual disability (ID), severe cognitive and behavioral impairments, potentially diagnosed as a comorbid autism spectrum disorder or attention-deficit hyperactivity disorder. Quality of life is ofte...

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Bibliographic Details
Main Authors: A. R. Müller, B. den Hollander, P. M. van de Ven, K. C. B. Roes, L. Geertjens, H. Bruining, C. D. M. van Karnebeek, F. E. Jansen, M. C. Y. de Wit, L. W. ten Hoopen, A. B. Rietman, B. Dierckx, F. A. Wijburg, E. Boot, M. M. G. Brands, A. M. van Eeghen
Format: Article
Language:English
Published: BMC 2024-01-01
Series:BMC Psychiatry
Subjects:
N-of-1
Cannabidiol
CBD
Tuberous sclerosis complex
Sanfilippo disease
Mucopolysaccharidosis
Online Access:https://doi.org/10.1186/s12888-023-05422-3

Internet

https://doi.org/10.1186/s12888-023-05422-3

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