Therapy for mucopolysaccharidosis type II with an intravenous blood-brain barrier-crossing enzyme (JR-141): Phase III global clinical trial design

Publication date: February 2021Source: Molecular Genetics and Metabolism, Volume 132, Issue 2Author(s): Sairei So, Paul Harmatz, Minako Kobayashi, Toshiaki Ikeda, Mariko Yamaoka, Tatsuyoshi Yamamoto, Teiji Tomio, Mathias Schmidt, Kazunori Tanizawa, Yuji Sato, Hiroyuki Sonoda
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research