FABRY DISEASE
Clinical trials for FABRY DISEASE explained in plain language.
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New study checks drug safety for fabry patients on dialysis
Disease control OngoingThis study looks at how safe migalastat is for people with Fabry disease who also have severe kidney issues or are on dialysis. About 14 adults will take the drug to see how it behaves in their bodies and if it causes side effects. The goal is to make sure the drug is used correc…
Matched conditions: FABRY DISEASE
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated May 01, 2026 16:00 UTC
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Fabry patients get extended look at enzyme therapy safety
Disease control OngoingThis study checks the long-term safety and effectiveness of a drug called pegunigalsidase alfa in adults with Fabry disease. All 29 participants had already completed a previous study of the same drug. They receive an infusion every 4 weeks for up to several years. The goal is to…
Matched conditions: FABRY DISEASE
Phase: PHASE3 • Sponsor: Chiesi Farmaceutici S.p.A. • Aim: Disease control
Last updated Apr 30, 2026 15:50 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
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Fabry disease patients try exercise as medicine in new study
Symptom relief ENROLLING_BY_INVITATIONThis study tests whether a 12-week physiotherapy and exercise program can improve physical fitness and quality of life in adults with Fabry disease. Fifteen participants will do regular exercise sessions with body weight, resistance bands, and increased daily activity. The goal i…
Matched conditions: FABRY DISEASE
Phase: NA • Sponsor: General University Hospital, Prague • Aim: Symptom relief
Last updated Apr 30, 2026 15:49 UTC
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Heart damage in fabry disease: new study tracks silent progression
Knowledge-focused OngoingThis study follows 31 Chinese adults with a specific genetic mutation (IVS4+919G>A) that causes Fabry disease, a condition where harmful substances build up and damage organs, especially the heart. Researchers use advanced heart scans and blood tests to track how the disease prog…
Matched conditions: FABRY DISEASE
Sponsor: Chinese University of Hong Kong • Aim: Knowledge-focused
Last updated May 01, 2026 15:59 UTC
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New york babies to get extra screening for rare diseases in landmark study
Knowledge-focused ENROLLING_BY_INVITATIONThis study offers parents of newborns in select New York hospitals the option to screen their baby for a panel of rare genetic disorders, beyond standard newborn screening. The goal is to learn how accurate these tests are, how common these conditions are, and whether early diagn…
Matched conditions: FABRY DISEASE
Sponsor: Albert Einstein College of Medicine • Aim: Knowledge-focused
Last updated Apr 30, 2026 15:51 UTC