FABRY DISEASE
Clinical trials for FABRY DISEASE explained in plain language.
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Hope for fabry patients: new drug shows promise in Long-Term trial
Disease control OngoingThis study looks at the long-term safety and effectiveness of a drug called pegunigalsidase alfa for adults with Fabry disease, a rare genetic condition that can damage organs. Participants receive an infusion every four weeks and are monitored for side effects, kidney function, …
Matched conditions: FABRY DISEASE
Phase: PHASE3 • Sponsor: Chiesi Farmaceutici S.p.A. • Aim: Disease control
Last updated Apr 28, 2026 21:35 UTC
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New fabry drug aims to shrink thickened heart muscle
Disease control OngoingThis study tests a new drug called venglustat against usual treatments for Fabry disease in adults who also have a thickened heart muscle (left ventricular hypertrophy). About 104 people aged 18 to 65 will take part for 18 months. The main goal is to see if venglustat can reduce …
Matched conditions: FABRY DISEASE
Phase: PHASE3 • Sponsor: Sanofi • Aim: Disease control
Last updated Apr 28, 2026 13:02 UTC