An adolescent boy was diagnosed with Sanfilippo syndrome type A after the onset of an acute and transient psychotic disorder,…
Articles by Steve Bryson, PhD
A newly identified mutation was found in a boy from Kosovo with Sanfilippo syndrome type B alongside a well-known…
The investigational, brain-penetrating enzyme replacement therapy DNL126 was found to correct neurological signs of Sanfilippo syndrome type A in…
Researchers identified 11 new candidate metabolite biomarkers in the urine of people with Sanfilippo syndrome, a study reported. Metabolomics,…
Neurological, behavioral, and MRI examinations of a group of children and adolescents with Sanfilippo syndrome in Brazil showed increased…
Brain implantation of genetically corrected stem cells restored NAGLU enzyme activity and reduced signs of Sanfilippo syndrome type B in…
Stem cell gene therapy in children with Sanfilippo syndrome type A was well-tolerated and generated high levels of SGSH,…
According to parents of children with Sanfilippo syndrome, developmental delays, hyperactivity, sleep disturbances, hearing loss, and recurrent infections were…
Sanfilippo syndrome type A was found to be the most predominant form of the rare genetic disorder in Korea…
A mouse model of Sanfilippo syndrome had persistent energy demands driven by activated heat-generating fat cells and impaired recycling…