Characterization of early markers of disease in the mouse model of mucopolysaccharidosis IIIB
Source: Journal of Neurodevelopmental Disorders
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
Late-onset mucopolysaccharidosis type IIIA mimicking Usher syndrome
Source: American Journal of Medical Genetics, Part A
Drosophila melanogaster models of MPS IIIC (Hgsnat-deficiency) highlight the role of glia in disease presentation
Source: Journal of Inherited Metabolic Disease
Filipin complex-reactive brain lesions: A cautionary tale
Source: Neuropathology and Applied Neurobiology
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