Neurology
3 drugs are in development for Pompe Disease.
- Companies
- 3
- Clinical trials
- 12
Prepared research question
Which programs lead the Pompe Disease pipeline—and what evidence could change the picture?
Traceable citations · Unknowns marked
Drug Pipeline by Phase
| Drug | Company |
|---|---|
| AT2221 | AMICUS THERAPEUTICS, INC. (FOLD) |
| Drug | Company |
|---|---|
| mze001 | Maze Therapeutics, Inc. (MAZE) |
| Drug | Company |
|---|---|
| atx-04 | AVENUE THERAPEUTICS, INC. (ATXI) |
About this indication
Pompe Disease context
Also known as: Acid Alpha Glucosidase Deficiency, Acid Alpha-Glucosidase Deficiencies, Acid Maltase Deficiency Disease, Alpha-Glucosidase Deficiencies, Alpha-Glucosidase Deficiency, Deficiencies, GAA, Deficiency Disease, Lysosomal alpha-1,4-Glucosidase, Deficiency of Alpha Glucosidase, Deficiency, GAA, Disease, Pompe's, GSD II, GSD2, GSD2s, Generalized Glycogenoses, Glycogen Storage Disease II, Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, Glycogenosis 2, Glycogenosis, Generalized, Lysosomal alpha 1,4 Glucosidase Deficiency Disease, Type II, Glycogenosis, Type IIs, Glycogenosis
Incidence
0.3 / 100K/yr
Frequently asked questions
How many drugs are in development for Pompe Disease?
BioSniper tracks 3 drugs in development for Pompe Disease across 3 companies.
How many clinical trials target Pompe Disease?
There are 12 clinical trials targeting Pompe Disease tracked by BioSniper.