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

Harmatz, Paul

A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease. [electronic resource] - Molecular genetics and metabolism 04 2018 - 488-494 p. digital

Publication Type: Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

1096-7206

10.1016/j.ymgme.2018.02.006 doi


Adolescent
Adult
Child
Female
Follow-Up Studies
Glucuronidase--administration & dosage
Humans
Male
Mucopolysaccharidosis VII--enzymology
Prognosis
Recombinant Proteins--administration & dosage
Young Adult