Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

计划入组 20 人 · 首次发布于 2018 年 8 月 2 日

N-sulfoglucosamine sulfohydrolase recombinant

Olenasufligene relduparvovec

N&N Pharmaceuticals, Inc.

United States

n-Lorem Foundation

United States

Allen N Wilkins MD

United States

Ultragenyx's UX111 Gene Therapy Shows Promise in Sanfilippo Syndrome Type A

Ultragenyx's UX111 Gene Therapy Shows Sustained Efficacy in MPS IIIA Patients

Denali Therapeutics Announces Positive Clinical Updates and Financial Results for Q1 2024