2022 Program: 7th Update on Fabry Disease:
Venue:
Program Steering Committee:
This educational activity is supported by unrestricted medical education grants from:
Major Sponsors:
Sponsors:
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, structured debates 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 conerns, 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 encourage, with hybrid presentations.
Preliminary Program
(updated Sept. 2, 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 TBA)
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 – 14:30 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)
14:30 – 15:00 Recent advances in Brain Imaging in Fabry Disease; TBA Andreas Fellgiebel (University of Mainz) pending respons
15:00 – 15:25 White matter lesions, depression, cognitive decline, and dementia in Fabry disease; Alessandro Burlina, MD) (San Bassiano Hospital)
15:25 – 15:50 Understanding Fabry associated pain—a translational approach ---Nurcan Üçeyler, MD (University of Würzburg)
15:50 – 16:15 Pain as Primary Endpoint in MODIFY Phase 3 trial of Lucerastat in Fabry disease (NCT03425539); Peter Nordbeck, MD (University of Würzburg)
16:15 – 16:45 Group discussion
16:45 – 17:30 COFFEE BREAK
Sesson 5: Gastro-Intestinal Issues and Fabry Disease (Chairs: Renzo Mignani, MD (Ospedale Infermi, Rimini) and Malte Lenders, PhD (University of Münster)
17:30 – 17:50 The Microbiome in Fabry disease; Alberto Ortiz, MD (Fundacion Jimenez Diaz)
17:50 – 18:10 Oral alpha-Galactosidase Therapy; Eva Brand, MD PhD (University of Münster)
18:10 – 18:30 Patient-Reported Outcomes for GI Complaints in Fabry Disease; Alan Shields, PhD (Adelphi Values, Boston)
18:30 – 19:00 Group discussion
Monday Evening: Free after Reception and Poster Viewing
Tuesday May 31st
Session 6 Inflammation, Autophagy and ROS in Fabry Disease (Chairs: Tobias Huber MD (University of Hamburg-Eppendorf) 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 7 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 8 Cardio-Renal Session (Chairs: Peter Nordbeck, MD (University of Würzburg) and Christoph Wanner, MD (University of Würzburg)
11:00 – 11:30 Renal Outcomes with ERT Treatment in Fabry Disease; David Warnock MD, (UAB))
11:30 – 12:00 Cardiac Outcomes with ERT Treatment in Fabry Disease; TBA Aleš Linhart, MD PhD
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 9 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