2022 Program: 7th Update on Fabry Disease:

Biomarkers, Progression and Treatment Opportunities
May 29-May 31, 2022: Würzburg Germany 
Supporting Organization: Kidneys for Life (Manchester, UK) 
Secretariat: Kongressmanagement bei Aey Congresse GmbH.  Berlin, Germany

Venue: Maritim Hotel and Conference Center, Würzburg Germany

Program Steering Committee:

Derralyn Hughes, Royal Free Hospital, London UK
Mirjam Langeveld, MD, AMC, Amsterdam NL
Albina Nowak, MD, University of Zurich, Zurich CH
Juan Politei, MD PhD, FESEN, Buenos Aires, AR
Andrew Talbot, Royal Melbourne Hospital, Melbourne AU
Stephen Waldek, Sunderland University UK
Christoph Wanner, University of Würzburg, Würzburg Germany
David G. Warnock, University of Alabama at Birmingham, USA
Michael West, Dalhousie University, Nova Scotia, Canada

This educational activity is supported by unrestricted medical education grants from: 

Major Sponsors:

Amicus Therapeutics

  Sponsors and Supporters: 

Idorsia Phsarmaceuticals Ltd, Protalix BioTherapeutics



The 1st Fabry Nephropathy Satellite "Focus on Fabry Nephropathy: Biomarkers, Progression and Disease Severity", an Official Satellite of the World Congress of Nephrology, was held in Bergamo Italy in May 2009. The second Fabry Nephropathy Satellite "Focus on Fabry Nephropathy: Biomarkers, Progression and Treatment Opportunities" was organized as an Official Satellite of the World Congress of Nephrology, and was held in Vancouver, Canada on April 12--14, 2011. The 3rd Update on Fabry Nephropathy was held in conjunction with the World Congress of Nephrology, in Hong Kong on June 4-5, 2013. The 4th Fabry Nephropathy Update was a Satellite of the ERA_EDTA 2015 Meeting, and was held in Manchester, UK on June 1-2, 2015. The 5th Fabry Nephropathy Update was an official Satellite of the 2017 ISN WCN Meeting, and was held in Mexico City, Mexico on April 25-27, 2017. The 6th Update on Fabry Disease was held in Prague, CZ, May 26-28, 2019. The 7th Update on Fabry Disease  will be held May 29-May 31, 2022 in Würzburg Germany


The 3-day conference features, state of the art plenary presentations, case discussions and poster presentations. The intended audience are content experts and "treaters" of Fabry Disease. The focus will be on emerging treatment approaches, baseline characteristics that determine the patient-specific responses to the treatment of Fabry disease, and antibody interactions with infused enzyme replacement therapy.


Because of COVID concerns, the meeting will be limited to approximately 80 face-to-face, fully vaccinated participants in a COVID-protected "bubble" at the Maritim Conference Center in Würzburg Germany.  A larger category of "virtual" participants, who will have live on-line access to the meeting, but will not be allowed to enter the "bubble". Poster submissions are encouraged, with hybrid presentations available.

Preliminary Program

(updated Nov. 20, 2021)

Sunday May 29th May

8 am On-site Registration

Workshop on Cell and Gene Therapy for Fabry Disease (Chairs: Michael West MD (Dalhousie University), and Yoshikatsu Eto, MD PhD (Jikei University)

 10:00 – 10:20            Update of AAV Approaches to Fabry Disease; Derralyn Hughes MD PhD (Royal Free Hospital)

 10:20 – 10:40            Update of Lenti-Virus Approaches to Fabry Disease; Kathy Nicholls, MD (Royal Melbourne Hospital)

 10:40 –11:10             CRISPR (Virtual Presentation): Knock-In and Knock-Out Approaches to Sickle Cell Disease; Mark C. Walters, MD (UCSF)

11:10 – 11:30             Group Discussion

11:30 – 12:45             Buffet Lunch

12:45 – 13:00 Welcome and Outline of Program: Christoph Wanner, MD and Peter Nordbeck, MD (University of Würzburg)

13:00 – 13:45             Keynote: Fabry disease-past, present and future; Derralyn Hughes MD PhD (Royal Free Hospital)

Session 1.                  New Approaches for Optimizing Treatment of Fabry Disease (Chairs: Dominique Germain, MD PhD, (Hôpital Raymond Poincaré)  and João Paulo Oliveira, MD PhD (University of Porto)

13:45 – 14:15             Directed Iterative Evolution of Enzymes for Treating Metabolic Diseases: Gjalt Huisman, PhD (Codexis, Inc)

14:15 – 14:45             Structure/Function Insights from Mutation Analysis of ERT: Scott Garman, PhD (University of Massachusetts)

14:45 – 15:15             PEGylation and Immunogenicity of ERT: David G. Warnock, MD (UAB)

15:15 – 15:45             Cardiomyocytes and Novel Treatment Approaches for Fabry Disease: James Shayman, MD (University of Michigan)

15:45 – 16:15             Group discussion

16:15 – 17:30             COFFEE BREAK [Networking]

17:30 – 19:00             Poster Session with Opening Reception (Conference Center:  Maritim Hotel Panorama Foyer)

19:30 – 21:30             Buffet Dinner for Face-to-Face Participants (Conference Center/Maritim Hotel)

Monday 30st May

Session 2.                    Artificial Intelligence, Machine Learning and Fabry Disease (Chairs:  John Jefferies, MD (University of Tennessee) and Javier A. Neyra, MD (University of Kentucky, Lexington )  

08:15 – 09:00             Keynote: Artificial Intelligence in the diagnosis of Fabry Disease: Joseph Zabinski, PhD (OM1, Inc.)

09:00 – 09:20             Artificial Intelligence, EKGs and Fabry Disease: Mehdi Namdar, MD (University of Geneva)

Session 3.         Biomarkers and Clinical Outcomes for Fabry Disease (Chairs: Kevin Mills, PhD (University College London), and Albina Nowak, MD (University of Zurich)

09:20 – 09:50               Molecular Basis, Diagnostics and Therapeutic Avenues for Fabry Disease: Prof. Dr. Hans Aerts (University of Leiden)

09:50 – 10:10               Organ specific biomarkers for Fabry Disease: Christiane Auray Blais, PhD (University of Sherbrooke)

10:10 – 10:30               Clinical and Biomarker Responses to Fabry Therapy: Daniel Bichet, MD (University of Montreal)

10:30 – 11:10             Group discussions

11:10 – 11:30             COFFEE BREAK [Networking and Poster Viewing]

11:30 –13:00    Difficult Cases: Series of Cases with Audience Response Before and After Presentations; Stephen Waldek, MD (University of  Sunderland) and Eric Wallace, MD (University of Alabama at Birmingham)

13:00 – 14:15    LUNCH buffet style – working lunch for poster viewing and networking.

Session 4.        Neurology and Fabry Disease (Chairs: Alessandro Burlina, MD (San Bassiano Hospital) and Juan Politei, MD (FESEN)

 14:15 – 14:35            Nociceptive behavior and central neuropeptidergic dysregulations in Fabry disease: Lessons from a Fabry mice model; Francesco Formaggio, PhD (Max Delbrück Center for Molecular Medicine Berlin)

14:35 – 14:55            White matter lesions, depression, cognitive decline, and dementia in Fabry disease; Alessandro Burlina, MD (San Bassiano Hospital)

14:55 – 15:15          Understanding Fabry associated pain—a translational approach ---Nurcan Üçeyler, MD (University of Würzburg) 

15:15 – 15:30          Group discussion

15:35 – 16:00             COFFEE BREAK

Sesson 5: Gastro-Intestinal Issues and Fabry Disease (Chairs: Renzo Mignani, MD (Ospedale Infermi, Rimini) and Malte Lenders, PhD (University of Münster)

16:00 – 16:20          The Microbiome in Fabry disease; Alberto Ortiz, MD (Fundacion Jimenez Diaz)

 16:20 – 16:40         Oral alpha-Galactosidase Therapy; Eva Brand, MD PhD (University of Münster)

16:40 – 17:00          Patient-Reported Outcomes for GI Complaints in Fabry Disease; Alan Shields, PhD (Adelphi Values, Boston)

17:00 – 17:20          Group discussion

Session 6: Substrate Reduction Therapy Workshop (Chairs: James Shayman, MD PhD (University of Michigan) and Christoph Wanner, MD (University of Würzburg)

17:30 – 17:50         Substrate Reduction Therapies and Fabry Disease; James Shayman, MD PhD (University of Michigan)

17:50 – 18:10         Clinical Trial Designs for Substrate Reduction Therapy; Christoph Wanner, MD (University of Würzburg)

18:10 – 18:30         Group discussion

19:00 – 20:30         Reception and Poster Viewing

                                 Monday Evening: Free after Reception and Poster Viewing


Tuesday May 31st

Session 7         Inflammation, Autophagy and ROS in Fabry Disease (Chairs: Paula Rozenfeld, MD  (CONICET-UNLP) and George Kaysen, MD PhD (UC Davis) 

08:00 – 08:30          Mitochondrial Structure, Function, and Turnover in Renal Epithelial Cells in Fabry Disease; Anke Schumann, MD PhD (University of Freiburg)

08:30 – 09:00         Novel Insights into Fabry Podocytopathy; Tobias Huber MD (University of Hamburg-Eppendorf) 

Session 8         Anti-drug antibodies (Chairs: Eva Brand, MD PhD (University of Münster) and Mirjam Langeveld, MD (AMC)

09:00 – 09:30          Impact of neutralizing antibodies on efficacy of ERT; Malte Lenders, PhD (University of Münster)

09:30– 09:45           Origin of IgE antibodies in patients naive to ERT; Andrew Talbot, MD (Royal Melbourne Hospital)

09:45 – 10:15          Clinical Implications and Treatment Modification for Fabry Patients with Positive ADA Status: Mirjam Langeveld, MD (AMC)

10:15 – 10:30          Group Discussion

10:30 – 11:00 COFFEE BREAK

Session 9       Cardio-Renal Session (Chairs: James Moon, MD (Barts Health NHS Trust) and Christoph Wanner, MD (University of Würzburg)

11:00 – 11:30            Renal Outcomes with ERT Treatment in Fabry Disease; Bojan Vujkovac, MD (General Hospital Slovenj Gradec)

11:30 – 12:00          Cardiac Outcomes with ERT Treatment in Fabry Disease; Peter Nordbeck, MD (University of Würzburg)

 12:00 – 12:30           Cardio-Renal Outcomes with Chaperone Therapy in Fabry Disease; Dominique Germain, MD PhD, (Hôpital Raymond Poincaré))

12:30 – 13:00            Optimizing Adjunctive Therapy in Cardio-Renal Disease: RAS and SGLT2 Inhibitors; Rajiv Agarwal, MD, (Indiana University) 

 12:00 – 12:30           Group discussion

13:30 – 14:30 LUNCH buffet style – working lunch for poster viewing and networking.

Session 10       Poster Session (Chair: Andrew Talbot, MD (Royal Melbourne Hospital) and Poster Judging Committee

14:30 – 15:30         4 x 10 minute presentations to poster prize winners preceded by prize presentations

 15:30 – 16:00            Closing Remarks