Program

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 https://www.maritim.de/de/hotels

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

The following entities provided financial sponsorship toward the logistical costs of the 7th International Update on Fabry Disease but have had no input into the agenda, content, or arrangements.

Chiesi Farmaceutici S.p.A, Idorsia Pharmaceuticals Ltd,

Sanofi Pharmaceuticals, Amicus Therapeutics

4D_Molecular Therapeutics, Sangamo Therapeutics

Protalix BioTherapeutics, AceLink Therapeutics, Freeline, uniQure NV

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.

Final Program

(updated May 14, 2022)

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             (Virtual Presentation): Update of Lenti-Virus Approaches to Fabry Disease; Kathy Nicholls, MD (Royal Melbourne Hospital)

10:40 –11:10              (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: Casey Hallows, 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)

Sunday Evening: Free after Reception and Poster Viewing

Monday 30st May

Session 2.                    Artificial Intelligence, Machine Learning and Fabry Disease (Chairs:  John Jefferies, MD (University of Tennessee) and Eric Wallace, MD (University of Alabama at Birmingham)  

08:00 – 08:30           Keynote: Artificial Intelligence in the Diagnosis of Fabry Disease: Joseph Zabinski, PhD (OM1, Inc.)

08:30 – 09:00             Artificial Intelligence, MRIs and Left Ventricular Mass Index in Fabry Disease: James C. Moon, MD PhD (Barts Hospital London)

09:00 – 09:20             Group Discussion

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:  Juan Politei, MD (FESEN) and Dawn Lanety (Emery University)

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

 16:20 – 16:40         Oral alpha-Galactosidase Therapy; Malte Lenders, PhD (University of Münster)

16:40 – 17:00          Group discussion

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

17:00 – 17:30         Substrate Reduction Therapies and Fabry Disease; James Shayman, MD PhD (University of Michigan)

17:30 – 17:45         Clinical Trial Designs for Substrate Reduction Therapy; Christoph Wanner, MD (University of Würzburg)

17:45 – 17:55          Pharmaco-dynamic Responses to Lucerastat in the Phase 3 MODIFY Trial; Peter Nordbeck, MD (University of Würzburg)

17:55 – 18:05          Pre-Clinical Results with a Novel Glucosylceramide Synthase Inhibitor (AL01211); Michael Babcock, PhD (ACELink Therapeutics)

18:05 – 18:15         A Study to Evaluate the Effect of Venglustat on Left Ventricular Mass Index in Adult Fabry Patients; James C. Moon, MD PhD (Bart’s Heart Center, London)

18:15 – 18:25         Rational Design of α-1,4-Galactosyltransferase Inhibitors as Substrate Reduction Therapy for the Treatment of Fabry Disease; Nicky de Koster,  (Leiden University, Leiden)

18:25 – 18:45         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 Sandro Feriozzi, MD (Belcolle Hospital)

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)

09:00 – 09:15          Group Discussion

Session 8         Anti-drug antibodies (Chairs: David Warnock, MD (University of Alabama at Birmingham) and Mirjam Langeveld, MD (AMC)

09:00 – 09:25          Impact of neutralizing antibodies on efficacy of ERT; Malte Lenders, PhD (University of Münster)

09:25 – 9:50          Clinical Implications and Treatment Modification for Fabry Patients with Positive ADA Status: Mirjam Langeveld, MD (AMC)

09:50 – 10:15          Group Discussion

10:15 – 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           Group discussion

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

Session 10       Poster Session (Chairs: Andrew Talbot, MD (Royal Melbourne Hospital) and Albina Nowak MD (University of Zurich with Poster Judging Committee

14:00 – 15:00         4 x 10 minute presentations to poster prize winners preceded by prize presentations

 15:00 – 15:15            Closing Remarks