The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII.
Wang, Raymond Y
The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII. [electronic resource] - Molecular genetics and metabolism 03 2020 - 219-227 p. digital
Publication Type: Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
1096-7206
10.1016/j.ymgme.2020.01.003 doi
Adolescent
Adult
Antibodies, Neutralizing
Blood-Brain Barrier--drug effects
Child
Cross-Over Studies
Enzyme Replacement Therapy
Female
Glucuronidase--administration & dosage
Glycosaminoglycans--urine
Humans
Male
Mucopolysaccharidosis VII--drug therapy
Rare Diseases--therapy
Treatment Outcome
The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII. [electronic resource] - Molecular genetics and metabolism 03 2020 - 219-227 p. digital
Publication Type: Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
1096-7206
10.1016/j.ymgme.2020.01.003 doi
Adolescent
Adult
Antibodies, Neutralizing
Blood-Brain Barrier--drug effects
Child
Cross-Over Studies
Enzyme Replacement Therapy
Female
Glucuronidase--administration & dosage
Glycosaminoglycans--urine
Humans
Male
Mucopolysaccharidosis VII--drug therapy
Rare Diseases--therapy
Treatment Outcome