FABRY DISEASE
Clinical trials for FABRY DISEASE explained in plain language.
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New study tracks Real-World effects of fabry drug in chinese patients
Disease control Recruiting nowThis study follows 200 Chinese children and adults with Fabry disease who are receiving Replagal (agalsidase alfa) as part of their regular care. Fabry disease is a rare inherited disorder that causes harmful fat buildup in blood vessels, damaging the heart, kidneys, and brain ov…
Matched conditions: FABRY DISEASE
Sponsor: Takeda • Aim: Disease control
Last updated Apr 28, 2026 21:36 UTC
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Gene therapy trial hopes to tame rare enzyme disorder
Disease control Recruiting nowThis early-stage study tests a single-dose gene therapy called AMT-191 in 12 adult men with classic Fabry disease. The main goal is to see if the treatment is safe and how it behaves in the body. Fabry disease is a rare genetic disorder that can cause pain, organ damage, and shor…
Matched conditions: FABRY DISEASE
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated Apr 28, 2026 13:02 UTC
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Rare stroke causes no longer overlooked: new italian network aims to end misdiagnosis
Knowledge-focused Recruiting nowThis study creates a network of hospitals across Italy to better understand and diagnose rare cerebrovascular diseases (rCVDs) like CADASIL, Fabry disease, and Moyamoya. Researchers will collect health information from 500 patients to describe the diseases' features and natural h…
Matched conditions: FABRY DISEASE
Sponsor: Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta • Aim: Knowledge-focused
Last updated Apr 28, 2026 21:36 UTC
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Heart scans reveal Drug's hidden power against rare disease
Knowledge-focused Recruiting nowThis study looks at whether a drug called agalsidase alfa can reduce inflammation in the hearts of people with Fabry disease. Researchers will use special heart scans (PET-CMR) to measure inflammation before and after 12 months of treatment. The study involves 25 adults with Fabr…
Matched conditions: FABRY DISEASE
Sponsor: Yonsei University • Aim: Knowledge-focused
Last updated Apr 28, 2026 13:02 UTC
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New study asks: is fabry treatment worth it for seniors?
Knowledge-focused Recruiting nowThis study looks at how Fabry disease affects the quality of life of people aged 65 and older, both those who take specific treatments and those who do not. Researchers will follow 100 participants for 5 years, using a standard quality-of-life questionnaire. The goal is to unders…
Matched conditions: FABRY DISEASE
Sponsor: Wladimir MAUHIN, Dr • Aim: Knowledge-focused
Last updated Apr 28, 2026 13:02 UTC