Treatment with resveratrol, a plant-derived compound that activates a cell cleansing process called autophagy, reduced the toxic buildup of heparan…
Articles by Marisa Horak, MS
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,…
Children with Sanfilippo syndrome have altered growth patterns — starting at birth, when infants with the genetic disorder are…
Tralesinidase alfa (AX 250), an experimental enzyme replacement therapy for Sanfilippo syndrome type B that’s now in clinical…
A short, intense course of eye movement desensitization reprocessing therapy, called EMDR, can help to lessen symptoms of trauma and…