A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease. [electronic resource]

By: Contributor(s): Producer: 20190708Description: 488-494 p. digitalISSN:
  • 1096-7206
Subject(s): Online resources: In: Molecular genetics and metabolism vol. 123
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Publication Type: Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

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