Lysosomal storage diseases
Clinical trials for Lysosomal storage diseases explained in plain language.
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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…
Matched conditions: LYSOSOMAL STORAGE DISEASES
Phase 1/2 • Sponsor: Spur Therapeutics • Aim: Cure
Last updated Sep 05, 2026 00:00 UTC
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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…
Matched conditions: LYSOSOMAL STORAGE DISEASES
Phase 1/2 • Sponsor: Spur Therapeutics • Aim: Cure
Last updated Sep 05, 2026 00:00 UTC