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Neurology

4 drugs are in development for Fabry Disease.

Companies
4
Clinical trials
24
BioSniper AIPage evidence connected

Prepared research question

Which programs lead the Fabry Disease pipeline—and what evidence could change the picture?

Run cited research

Traceable citations · Unknowns marked

Drug Pipeline by Phase

Approved(1)
Preclinical(1)
Phase 1/Phase 2(2)
DrugCompany
amt-191uniQure N.V. (QURE)
isaralgagene civaparvovecSANGAMO THERAPEUTICS, INC (SGMOQ)
About this indication

Fabry Disease context

Neurology·MeSH: Fabry Disease (D000795)

Also known as: Anderson Fabry Disease, Angiokeratoma Corporis Diffusum, Angiokeratoma Diffuse, Deficiency, Ceramide Trihexosidase, Deficiency, GLA, Deficiency, alpha-Galactosidase A, Diffuse Angiokeratoma, Fabry's Disease, Lipidosis, Hereditary Dystopic, alpha Galactosidase A Deficiency Disease

Incidence

0.2 / 100K/yr

Frequently asked questions

How many drugs are in development for Fabry Disease?

BioSniper tracks 4 drugs in development for Fabry Disease across 4 companies.

How many clinical trials target Fabry Disease?

There are 24 clinical trials targeting Fabry Disease tracked by BioSniper.