Fabry disease
MONDO:0010526Fabry disease (FD) is a progressive, inherited, multisystemic lysosomal storage disease characterized by specific neurological, cutaneous, renal, cardiovascular, cochleo-vestibular and cerebrovascular manifestations.
Also known as: Alpha-galactosidase A deficiency, Anderson-Fabry disease, FD, Fabry disease, Fabry's disease, Fd, angiokeratoma corporis diffusum, diffuse angiokeratoma
64 clinical trials for this condition and its sub-types, 64 tagged with Fabry disease itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →-
Can a One-Time gene therapy fix fabry disease for years?
Cure Stopped earlyThis study follows people with Fabry disease who have already received FLT190, an experimental gene therapy that delivers a working copy of the GLA gene. The goal is to see how safe the treatment is over the long term and whether its effects last. Researchers will track participa…
Phase 1/2 • Sponsor: Spur Therapeutics • Aim: Cure
Last updated Sep 05, 2026 00:00 UTC
-
Can a single gene infusion rewrite the story of fabry disease?
Cure Stopped earlyThis trial is testing a gene therapy called FLT190 in adult men with classic Fabry disease, a genetic condition that causes harmful fat buildup in cells. The therapy uses a modified virus to deliver a working copy of the faulty gene, potentially enabling the body to produce the m…
Phase 1/2 • Sponsor: Spur Therapeutics • Aim: Cure
Last updated Sep 05, 2026 00:00 UTC
-
Home infusions may help patients stick to treatment
Knowledge-focused Stopped earlyThis study looks at whether people with Fabry, Gaucher, or Hunter disease are more likely to continue their IV treatment when it's given at home versus at a hospital. Researchers will review existing data from 222 patients in Mexico. No new treatments are given; the goal is to un…
Sponsor: Takeda • Aim: Knowledge-focused
Last updated Sep 13, 2026 00:00 UTC
-
Fabry disease sperm study halted early
Knowledge-focused Stopped earlyThis study aimed to find out how common sperm problems are in men with Fabry disease. Researchers planned to check sperm samples from 22 men aged 18 to 65. The study was stopped early, so results are limited.
Sponsor: University Hospital, Bordeaux • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC