University College London
London, United Kingdom
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Fabry disease
What is new in Fabry disease research, which clinical trials are open, and who works on it. From public registries and PubMed, with the source and date of every fact.
Cure status
- Agalsidase alfaPhase 3 · NCT05280548
- Agalsidase beta (GZ419828)Phase 3 · NCT05280548
- LucerastatPhase 3 · NCT07778667
- MigalastatPhase 3 · NCT05280548
- migalastat HCl 150 mgPhase 3 · NCT04020055
- Migalastat HCl 20 mgPhase 3 · NCT06904261
- Venglustat (GZ402671)Phase 3 · NCT05206773 · NCT05280548
- PRX-102 1 mg/kg every 2 weeksPhase 2/3 · NCT05710692
- PRX-102 1 mg/kg every two weeksPhase 2/3 · NCT06328608
- PRX-102 2 mg/kg every 4 weeksPhase 2/3 · NCT05710692
- 4D-310Phase 1/2 · NCT04519749 · NCT05629559
- AMT-191Phase 1/2 · NCT06270316
- EXG110 InjectionPhase 1/2 · NCT06819514
- HM15421/GC1134APhase 1/2 · NCT06858397
- BBM-F101 injectionEarly phase 1 · NCT06207552
Closest to patients
- Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry DiseaseStudy to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry DiseaseChiesi Farmaceutici S.p.A. (sponsor) · 10 hospitals in JapanPhase 2/3Results expected 2027
- A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA VariantsA Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA VariantsHospital Universitario de la Paz · Madrid, Spain + 11 more hospitalsPhase 3Results expected 2027
- A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry DiseaseA Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry DiseaseGemeinnuetzige Salzburger Landeskliniken Betriebsgesellschaft mbH · Salzburg, Austria + 11 more hospitalsPhase 2/3Results expected 2028
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Latest news
Results, new trials and publications, newest first. Every item links to its source.
Results and new studies, newest first, in plain words.
Plain-language summary pending. Original title shown.
Publication
Impact of Anti-drug Antibodies in Patients with Fabry Disease under Enzyme Replacement Therapy: Long-Term Outcomes.
Impact of Anti-drug Antibodies in Patients with Fabry Disease under Enzyme Replacement Therapy: Long-Term Outcomes.
BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy
PubMed · PMID 42983075Publication
Early mitophagy defects and impaired mitochondrial energy metabolism drive target organ damage progression: Lessons from the Fabry heart.
Early mitophagy defects and impaired mitochondrial energy metabolism drive target organ damage progression: Lessons from the Fabry heart.
Cell reports
PubMed · PMID 42945215Observational study
Disease severity and lyso-Gb3 are associated with early echocardiographic markers of cardiac involvement in Fabry disease: a single-center observational study.
Disease severity and lyso-Gb3 are associated with early echocardiographic markers of cardiac involvement in Fabry disease: a single-center observational study.
Orphanet journal of rare diseases
PubMed · PMID 42947193Publication
Peripheral and cardiac determinants of exercise intolerance in classic Anderson-Fabry disease: insights from cardiopulmonary exercise testing and advanced echocardiography.
Peripheral and cardiac determinants of exercise intolerance in classic Anderson-Fabry disease: insights from cardiopulmonary exercise testing and advanced echocardiography.
Orphanet journal of rare diseases
PubMed · PMID 42947138
Organisations that can help
Helplines, patient groups and support services for you and your family.
Select your country to see the organisations there, starting with the ones closest to you.
Umbrella organisations (they connect national groups)
European Patients' Forum (EPF)
Umbrella of European patient organisations and national patient coalitions.
Visit site: European Patients' Forum (EPF)
EURORDIS – Rare Diseases Europe
Alliance of over 1,000 rare disease patient organisations, with a list of rare disease helplines by country.
Visit site: EURORDIS – Rare Diseases Europe
Fabry International Network
Global network of Fabry patient groups: resources guide, young adults community, humanitarian treatment programmes, webinars, help finding a national patient group.
Visit site: Fabry International Network
International Alliance of Patients' Organizations
Global alliance of patient organisations of all diseases, promoting patient-centred healthcare.
Visit site: International Alliance of Patients' Organizations
Frontlight is not affiliated with these organisations; we list them because they help.
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Find fellow researchers
Groups with active Fabry disease work.
University of Naples Federico II
Naples, Italy
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Royal Free London NHS Foundation Trust
London, United Kingdom
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Emory University
Atlanta, United States
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University of Bologna
Bologna, Italy
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Universitätsklinikum Würzburg
Würzburg, Germany
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Taipei Veterans General Hospital
Taipei, Taiwan
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Charles University
Prague, Czechia
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University of Florence
Florence, Italy
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IRCCS Azienda Ospedliero-Universitaria di Bologna Policlinico di Sant'Orsola
Bologna, Italy
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University of Padua
Padua, Italy
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Agostino Gemelli University Polyclinic
Rome, Italy
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