Scientific Programme
International conference on Advances in the Treatment of Fabry Disease
Prague A-D
Friday 16.05.2025
09:00
-
09:05
Welcome address
Aleš Linhart
09:05
-
10:00
Session 1: Fabry disease in the context of cardiomyopathies
Differential diagnosis of a hypertrophied heart
Perry M. Elliott /London/
Cardiac amyloidosis – differential diagnosis and treatments
Pablo García Pavía /Madrid/
Hypertrophic cardiomyopathy management in the era of myosin inhibitors
Iacopo Olivotto /Firenze/
10:20
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11:40
Session 2: Fabry cardiomyopathy – advances in diagnosis and management
Contributions of cardiac MRI to the understanding of Fabry disease
James Moon /London/
Novel insights from imaging studies
Elena Biagini /Bologna/
Obstructive cardiomyopathy in Fabry disease
Josef Marek /Praha/
ECG in Fabry cardiomyopathy – a forgotten method?
Mehdi Namdar /Genève/
12:00
-
12:30
Industry Symposium 1
12:30
-
13:00
Industry Symposium 2
14:00
-
15:20
Session 3: Fabry disease and the kidney
The role of renal biopsy in the understanding of pathophysiology and treatment effects
Camilla Tøndel /Bergen/
Can we improve outcomes by SGLT2 inhibitors RAAS blockers?
Christoph Wanner /Würzburg/
Monitoring kidney disease – is serum creatinine and eGFR just enough?
David Warnock
End-stage kidney disease – what next? How do patients do on Haemodialysis and after kidney transplantation. Does the immune response to ERT differ?
Sima Canaan-Kühl /Berlin/
The role of podocytes and treatment effects
Alberto Ortiz /Madrid/
15:40
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17:00
Session 4: Advancements in understanding mechanisms of the disease – beyond the storage
The role of alpha-synuclein in the pathogenesis of Fabry Nephropathy
Fabian Braun /Hamburg/
The role of mitochondrial dysfunction and muscular abnormalities in Fabry disease
Guido Iaccarino /Napoli/
Endoplasmic reticulum and its role in cellular function
Moran Dvela Levitt /Ramat Gan/
Agalopathy – the role of ER stress in pathophysiology of Fabry disease
Martina Živná /Praha/
17:00
-
18:00
Session 5: Setting-up a multidisciplinary team of a Fabry center
Paediatric Fabry disease – the transitional care model issue
Federico Pieruzzi /Monza/
Organization of a multidisciplinary Fabry center team
Bojan Vujkovac /Slovenj Gradec/
“Once upon a time in Prague... there was a...?”
Gabriela Dostálová /Praha/
18:00
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18:05
Day 1 Adjourn
Aleš Linhart
Prague A-D
Saturday 17.05.2025
08:40
-
10:00
Session 6: Current and novel therapies – where do we stand?
Switch from agalsidase beta to migalastat – registry data
Antonio Pisani /Napoli/
Migalastat – analysis of registry data, amenability testing, clinical and biomarker monitoring
Daniel G. Bichet /Montréal/
Substrate reduction therapies – where do we stand?
Dominique P. Germain /Versailles/
Update on gene therapies in Fabry disease
Raphael Schiffmann /Emeryville, CA/
Enzyme-replacement therapies – long term data
Aleš Linhart /Praha/
10:20
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11:40
Session 7: Monitoring of the disease and treatments impacts
Monitoring PROs in Fabry disease
Uma Ramaswami /London/
Measurements of migalastat levels and antibody responses
Malte Lenders /Muenster/
Antibody response to enzyme replacement therapies
Maud Janssens /Amsterdam/
Lyso Gb3 and novel biomarkers
Albina Nowak /Zürich/
12:00
-
12:30
Industry Symposium 3
12:30
-
13:00
Industry Symposium 4
14:00
-
14:50
Split-out abstract sessions
15:00
-
16:20
Session 8: Advancements in understanding mechanisms of the disease and treatment effects
Peroxidasin (PXDN) expression in kidney tissue of patients carrying GLA gene variants, with or without Fabry
João Paulo Oliveira /Porto/
The role of inflammation in pathogenesis of cardiac involvement
Maurizio Pieroni /Firenze/
Cerebrovascular events in Fabry patients - clinical course and outcomes
Alessandro Burlina /Bassano del Grappa/
Gastrointestinal complications in Fabry patients
Juan Politei /Buenos Aires/
16:40
-
18:00
Session 9: Genetics, late onset variants, disease management and outcomes
Novel insights in Fabry genetics
Gheona Altarescu /Jerusalem/
How to read genetic test results conflicting with clinical evidence
Stanislav Kmoch /Praha/
Fabry disease due to p.F113L mutation
Olga Azevedo /Guimarães/
Fabry disease due to p.N215S mutation
Ana Jovanovic /Salford/
18:00
-
18:10
Young investigators Awards
18:10
-
18:15
Day 2 Adjourn
Dominique P. Germain
