TY  - GEN
AU  - Harmatz,Paul
AU  - Whitley,Chester B
AU  - Wang,Raymond Y
AU  - Bauer,Mislen
AU  - Song,Wenjie
AU  - Haller,Christine
AU  - Kakkis,Emil
TI  - A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease
SN  - 1096-7206
PY  - 2019///0708
KW  - Adolescent
KW  - Adult
KW  - Child
KW  - Female
KW  - Follow-Up Studies
KW  - Glucuronidase
KW  - administration & dosage
KW  - Humans
KW  - Male
KW  - Mucopolysaccharidosis VII
KW  - enzymology
KW  - Prognosis
KW  - Recombinant Proteins
KW  - Young Adult
N1  - Publication Type: Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
UR  - https://doi.org/10.1016/j.ymgme.2018.02.006
ER  -