Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Pompe Disease - Overview
Pompe Disease - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Pompe Disease - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Pompe Disease - Companies Involved in Therapeutics Development
Pompe Disease - Drug Profiles
Pompe Disease - Dormant Projects
Pompe Disease - Discontinued Products
Pompe Disease - Product Development Milestones
Featured News & Press Releases
Aug 11, 2022: FDA grants Orphan Drug status to Aro Biotherapeutics’ Pompe disease therapy
Jun 28, 2022: Nexviadyme (avalglucosidase alfa) approved by European Commission as a potential new standard of care for the treatment of Pompe Disease
May 18, 2022: AVROBIO announces preclinical gene therapy data for Pompe Disease at American Society of Gene and Cell Therapy (ASGCT) Annual Meeting
May 11, 2022: Aro Biotherapeutics to present new preclinical data highlighting the potential of Centyrin-siR conjugates for the treatment of Pompe disease
May 10, 2022: Amicus Therapeutics receives notification of PDUFA date extensions for AT-GAA
Mar 16, 2022: Amicus Therapeutics announces positive long-term data from phase 1/2 study of AT-GAA in Pompe Disease at the 2022 MDA Clinical & Scientific Conference
Mar 10, 2022: Amicus Therapeutics announces presentation and posters at the 2022 MDA Clinical & Scientific Conference
Feb 18, 2022: Maze Therapeutics initiates dosing in Phase I trial of Pompe disease asset
Feb 10, 2022: M6P Therapeutics presents promising preclinical data in Pompe disease at the 18th Annual WORLDSymposium 2022
Feb 10, 2022: Maze Therapeutics presents new preclinical data supporting advancement of MZE001 as a potential treatment for Pompe disease
Feb 08, 2022: Nexviazyme (avalglucosidase alfa) shows sustained improvements in respiratory function and mobility in patients with Pompe disease
Jan 31, 2022: Maze Therapeutics to present preclinical data highlighting MZE001 as a potential treatment for Pompe disease at 2022 WORLD Symposium
Dec 03, 2021: European Medicines Agency validates Amicus Therapeutics marketing authorization applications for AT-GAA for the treatment of Pompe disease
Nov 29, 2021: Sanofi’s Pompe Disease drug Nexviazyme now available in Japan
Nov 18, 2021: The Lancet Neurology publishes pivotal Phase 3 PROPEL study results of AT-GAA in late-onset Pompe disease
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures

List of Figures
Number of Products under Development for Pompe Disease, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Number of Products by Targets, 2022
Number of Products by Stage and Targets, 2022
Number of Products by Mechanism of Actions, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Routes of Administration, 2022
Number of Products by Stage and Routes of Administration, 2022
Number of Products by Molecule Types, 2022
Number of Products by Stage and Molecule Types, 2022

List of Tables

List of Tables
Number of Products under Development for Pompe Disease, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Products under Development by Universities/Institutes, 2022
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Pompe Disease - Dormant Projects, 2022
Pompe Disease - Discontinued Products, 2022