The case of a Mexican youth with Sanfilippo syndrome type B who presented with atypical symptoms of the disease,…
Articles by Marta Figueiredo, PhD
Most children with Sanfilippo syndrome type B show predictable downward trajectories in cognitive function, behavior, and brain volume that…
Ultragenyx Pharmaceutical has closed an agreement giving it global licensing, manufacturing, and commercialization rights to UX111 (formerly ABO-102), an…
Complications shortly after birth and a slow disease progression — combined with poor disease awareness — delayed a correct diagnosis…
Brain-in-a-Dish, a research project that aims to develop patient-derived cell models to study Sanfilippo syndrome and identify potential treatments,…
The European Commission has granted an orphan drug designation to JR-441, JCR Pharmaceuticals’ brain-penetrating experimental enzyme replacement therapy (ERT)…
Lysogene is terminating its licensing agreement with Sarepta Therapeutics for LYS-SAF302, its investigational gene therapy for…
Scientists have discovered a potential novel approach to possibly correct the neurological features of Sanfilippo syndrome type B and…
Recent advances in treating people with Sanfilippo syndrome and other mucopolysaccharidoses (MPS) were among topics addressed at the 16th International…
A single high dose of ABO-102, Abeona Therapeutics’ experimental gene therapy, prevented disease-associated brain shrinkage in three young children…