A hematopoietic stem and progenitor cell (HSPC) transplant eased several disease symptoms, including motor problems and enlarged organs, in a…
Articles by Lindsey Shapiro, PhD
Ultragenyx Pharmaceutical has submitted a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated…
The release of extracellular vesicles (EVs) from microglia may play a key role in driving neurodegeneration in Sanfilippo syndrome.
A number of proteins that accumulate to toxic levels in other neurodegenerative diseases also build up in cells derived from…
A carbohydrate molecule derived from marine bacteria was able to reduce inflammation in the brain, reverse metabolic dysfunction, and preserve…
Tralesinidase alfa, an experimental enzyme replacement therapy for people with Sanfilippo syndrome type B, was safe and showed…
An experimental enzyme replacement therapy (ERT) called tralesinidase alfa (AX 250) was found in a clinical trial to safely…
Children with Sanfilippo syndrome have impaired growth compared with the general population, but these deviations are milder than those…
The antidepressant fluoxetine was able to improve cognition and reduce the buildup of toxic molecules in a mouse model of…
Researchers from the Canadian Glycomics Network (GlycoNet) are developing a new combination therapy that’s showing promising preclinical results for…