A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease
Harmatz, Paul, Whitley, Chester B., Wang, Raymond Y., Bauer, Mislen, Song, Wenjie, Haller, Christine, Kakkis, EmilLanguage:
english
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2018.02.006
Date:
February, 2018
File:
PDF, 307 KB
english, 2018