Light-sensing cells in the eyes become damaged and die in Sanfilippo syndrome type C before the onset of behavioral…
Articles by Marisa Wexler, MS
The Lysosomal and Rare Disorders Research and Treatment Center is recruiting people with Sanfilippo syndrome for an observational…
Treatment with sugary molecules called iminosugars can reduce the toxic buildup of heparan sulfate in cellular models of Sanfilippo…
Treatment with resveratrol, a plant-derived compound that activates a cell cleansing process called autophagy, reduced the toxic buildup of heparan…
A novel enzyme replacement therapy developed by BioMarin Pharmaceutical for Sanfilippo syndrome type A showed promising effects in…
An international group of experts has published a set of consensus guidelines outlining how best to provide medical care for…
Children with Sanfilippo syndrome type A who were treated with the investigational gene therapy LYS-SAF302 before 2.5 years of age…
A never-before-reported mutation in the gene NAGLU was found to cause Sanfilippo syndrome type B in an 8-year-old girl in…
Later this month, the Czech government will host a conference where Deputy Minister of Health Jakub Dvořáček will present a…
When assessing bone density in people with conditions like Sanfilippo syndrome, it is necessary to account for impaired growth,…