- Article
Personalized Drug Repurposing Screen Identifies Patient-Specific Therapeutic Candidates for Mucopolysaccharidosis Type IIIB
- Kathleen D. McDaniel,
- Neda Ghousifam,
- Rodney A. Bowling,
- Catherine Z. Chen,
- Wei Zheng and
- Zeenat A. Shyr
Background: Mucopolysaccharidosis type IIIB (MPSIIIB, Sanfilippo syndrome type B) is a rare lysosomal storage disease caused by deficiency of alpha-N-acetylglucosaminidase (NAGLU) enzyme, leading to progressive accumulation of heparan sulfate and sev...

