Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease

首次发布于 2003 年 12 月 25 日

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

计划入组 16 人 · 首次发布于 2005 年 8 月 2 日

Pompe Telemedicine Developmental Study

计划入组 22 人 · 首次发布于 2016 年 11 月 1 日

Pompe disease(Codexis)

AOC-Pompe disease

Gene therapy (AAV, Pompe disease)

Azienda Ospedaliera Ospedale Infantile Regina Margherita Sant Anna

Italy

Rare Disease Research, LLC

CASTLEMAN DISEASE COLLABORATIVE NETWORK

United States

Sanofi's Nexviazyme Meets All Endpoints in Phase III Baby-COMET Trial for Infantile-Onset Pompe Disease

GeneVentiv Therapeutics Receives FDA Alignment on Gene Therapy Development Plan for Hemophilia A

FDA Grants Breakthrough Therapy Designation to Adrabetadex for Infantile-Onset Niemann-Pick Disease Type C