TY  - JOUR
AU  - Lau, Heather A
AU  - Viskochil, David
AU  - Tanpaiboon, Pranoot
AU  - Lopez, Antonio Gonzalez-Meneses
AU  - Martins, Esmeralda
AU  - Taylor, Julie
AU  - Malkus, Betsy
AU  - Zhang, Lin
AU  - Jurecka, Agnieszka
AU  - Marsden, Deborah
PY  - 2022
DO  - 10.1016/j.ymgme.2022.03.002
UR  - http://hdl.handle.net/10668/22551
T2  - Molecular genetics and metabolism
AB  - Mucopolysaccharidosis (MPS) VII is an ultra-rare, autosomal-recessive, metabolic disease caused by a deficiency of β-glucuronidase, a lysosomal enzyme that hydrolyzes glycosaminoglycans (GAGs), including dermatan sulfate (DS), chondroitin sulfate, and...
LA  - en
KW  - Growth
KW  - Hepatosplenomegaly
KW  - Mucopolysaccharidosis VII
KW  - Pediatric patients.
KW  - Urinary glycosaminoglycan
KW  - Vestronidase alfa
KW  - Child
KW  - Enzyme Replacement Therapy
KW  - Glucuronidase
KW  - Glycosaminoglycans
KW  - Hepatomegaly
KW  - Humans
KW  - Hydrolases
KW  - Mucopolysaccharidosis VII
KW  - Splenomegaly
TI  - Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII.
TY  - research article
VL  - 136
ER  -