Wang, Raymond Y <br/><br/>
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 
<br/><br/> Publication Type: Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't 
<br/><br/><label>ISSN: </label>1096-7206 
<br/><br/><label>Standard No.: </label>10.1016/j.ymgme.2020.01.003  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Adolescent<br/>Adult<br/>Antibodies, Neutralizing<br/>Blood-Brain Barrier--drug effects<br/>Child<br/>Cross-Over Studies<br/>Enzyme Replacement Therapy<br/>Female<br/>Glucuronidase--administration & dosage<br/>Glycosaminoglycans--urine<br/>Humans<br/>Male<br/>Mucopolysaccharidosis VII--drug therapy<br/>Rare Diseases--therapy<br/>Treatment Outcome