Two types of nerve cells, namely neurons and astrocytes, with Sanfilippo syndrome-like characteristics have been created by converting stem cells with disease-associated mutations, according to a study from researchers in Europe. Compared to cell lines currently used in Sanfilippo…
Investigational enzyme replacement therapy BMN-250 successfully delivers the NAGLU enzyme — present in low levels in Sanfilippo type B patients — to key cells of the brain using different cellular pathways, a study has found. The study, “BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different…
The first fly model of Sanfilippo syndrome type A has been developed and characterized, providing new data on genes involved in disease development. The study, “Neuronal-specific impairment of heparan sulfate degradation in Drosophila reveals pathogenic mechanisms for Mucopolysaccharidosis type IIIA,” appeared in the journal Experimental Neurology. Sanfilippo…
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