Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII
Sign Up to like & getrecommendations! Published in 2021 at "Molecular Genetics and Metabolism Reports"
DOI: 10.1016/j.ymgmr.2021.100774
Abstract: Vestronidase alfa is an enzyme replacement therapy for mucopolysaccharidosis VII (MPS VII). In this open-label, phase 1/2 study, three subjects with MPS VII received intravenous vestronidase alfa administered every other week (QOW) for 14 weeks… read more here.
Keywords: mucopolysaccharidosis vii; open label; label phase; vestronidase ... See more keywords
Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome)
Sign Up to like & getrecommendations! Published in 2022 at "Therapeutics and Clinical Risk Management"
DOI: 10.2147/tcrm.s351300
Abstract: Abstract Mucopolysaccharidosis VII (MPS VII, Sly syndrome) is an ultra-rare lysosomal disease caused by a deficiency of the enzyme β-glucuronidase (GUS). The diagnosis is suspected based on a range of symptoms that are common to… read more here.
Keywords: diagnosis; sly syndrome; vii sly; mucopolysaccharidosis vii ... See more keywords