Extracellular vesicles from microglial cells activated by abnormal heparan sulfate oligosaccharides from Sanfilippo patients impair neuronal dendritic arborization
Source: Molecular Medicine
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
Evaluation of neuroretina following i.v. or intra-CSF AAV9 gene replacement in mice with MPS IIIA, a childhood dementia
Source: CNS Neuroscience and Therapeutics
Mucopolysaccharidosis (MPS IIIA) mice have increased lung compliance and airway resistance, decreased diaphragm strength, and no change in alveolar structure
Source: American Journal of Physiology - Lung Cellular and Molecular Physiology
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