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
Source: BMC Psychiatry
Filipin complex-reactive brain lesions: A cautionary tale
Source: Neuropathology and Applied Neurobiology
Clinical and Molecular Characterization of Mucopolysaccharidosis Type 3A and 3B in a Turkish Series
Source: Molecular Syndromology
Drosophila melanogaster models of MPS IIIC (Hgsnat-deficiency) highlight the role of glia in disease presentation
Source: Journal of Inherited Metabolic Disease
Ability change across multiple domains in mucopolysaccharidosis (Sanfilippo syndrome) type IIIA
Source: Molecular Genetics and Metabolism
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