Day 1 / Oct. 2 (FRI)
9:00-9:30
- Chair
- Robert J. Desnick (Icahn School of Medicine at Mount Sinai, USA)
Opening and New proposal of Fabry disease after NBS
Yoshikatsu Eto (Southern Tohoku Institute for Neuroscience / The Jikei University School of Medicine, Japan)

9:30-10:30
Unique Case Presentations of Fabry disease (15 min x 4)
- Chair
- Aya Narita (Iseikai International General Hospital, Japan)
Masahisa Kobayashi (The Jikei University School of Medicine, Japan)
1) Rescue from sudden ventricular tachycardia and fibrillation using wearable cardioverter-defibrillator in male late-onset Fabry disease patient
Toraaki Okuyama (The Jikei University School of Medicine, Japan)
2) Reduced recurrence of strokes after switching to agalsidase beta in Fabry disease: two case reports
Yoshimi Fujita (The Jikei University School of Medicine, Japan)
3) Clinical Decision Making for Diagnosis and Treatment Initiation in Late Onset Mild Fabry Disease
Kaoru Eto (Tokyo Women’s Medical University Adachi Medical Center, Japan)
4) SGLT2 Inhibitors in Fabry Disease: Evidence of Renoprotection from Case-Based and Clinical Analyses
Hayaki Okamoto (Kobe University Graduate School of Medicine, Japan)
10:40-11:20(Co-sponsored by Sanofi)
- Chair
- Yoshikatsu Eto (Southern Tohoku Institute for Neuroscience / The Jikei University School of Medicine, Japan)
Fabry Disease: Major Advances in Delineation, Diagnosis and Treatment
Robert J. Desnick (Icahn School of Medicine at Mount Sinai, USA)
11:20-12:20(Co-sponsored by Sumitomo Pharma)
Key Points on Fabry Disease and LysoGb3
- Chair
- Motomichi Kosuga (National Center for Child Health and Development, Japan)
Kazuya Tsuboi (Nagoya Central Hospital, Japan)
1) Biomarkers in Fabry disease: Diagnosis and treatment monitoring
Takahiro Tsukimura (Meiji Pharmaceutical University, Japan) - 30 min
2) A biochemical and pharmacological comparison of three agalsidases approved and used in Japan
Hitoshi Sakuraba (Meiji Pharmaceutical University, Japan) - 30 min
12:30-13:10 Luncheon Seminar (Co-sponsored by JCR)
- Chair
- Torayuki Okuyama (Saitama Medical University, Japan)
Fabry Disease: Real World Evidence (RWE) of Treatment Outcomes
Roberto Giugliani (Federal University of Rio Grande do Sul, Brazil) - 40 min
13:20-13:50
New Therapy
- Chair
- Dau-Ming Niu (National Yang Ming Chiao Tung University, Taiwan)
Splicing Modulation Therapy for Taiwanese Cardiac Fabry Disease (GLA c.639+919G>A)
Tomonari Awaya (Osaka Metropolitan University Graduate School of Medicine, Japan) - 30min
13:50-14:30
- Chair
- Yukio Yuzawa (Vice Chairman, Fujita Academy, Japan)
Second Generation ERTs for Fabry Disease
David G. Warnock (The University of Alabama at Birmingham, USA) - 40 min
14:30-15:40
Organ Specific Outcomes - Kidney, Heart and Brain
1. Renal Problems
- Chair
- David G. Warnock (The University of Alabama at Birmingham, USA)
Ohsuke Migita (St. Marianna University School of Medicine, Japan)
1) The Evolving Landscape of Fabry Nephropathy
Naoki Nakagawa (Asahikawa Medical University, Japan) - 30 min
2) Increasing knowledge and tools within Fabry nephropathy
Camilla Tøndel (Univ of Bergen, Norway)(Web) - 40 min
15:50-17:20(Co-sponsored by Sanofi)
- Chair
- Hiroyuki Yamakawa (Keio University School of Medicine, Japan)
Katsuya Nakamura (Shinshu University School of Medicine, Japan)
2. Cardiovascular / CNS events
1) Cardiac Involvement in Fabry Disease: Clinical and Pathological Insights
Koji Higuchi (National Institute of Fitness and Sports in Kanoya, Japan)
2) Non-invasive Assessment of Cardiac Involvement in Fabry Disease Using Multimodality Imaging
Kenichi Hongo (The Jikei University School of Medicine, Japan) - 30 min
3) Fabry Disease and Stroke
Alessandro Burlina (St. Bassiano Hospital, Italy) (Web) - 30 min
17:20-18:00
- Chair
- Takeo Iwamoto (Southern Tohoku Institute for Neuroscience /The Jikei University School of Medicine, Tokyo)
Roberto Giugliani (Federal University of Rio Grande do Sul, Brazil)
LysoGb3 as a Biomarker in Fabry disease
Albina Nowak (University Zurich, Switzerland) - 40 min
18:00-18:40
- Chair
- Hiroyuki Ida (The Jikei University School of Medicine, Japan)
Antidrug antibodies in Fabry disease: Impact and challenge
Malte Lenders (Interdisciplinary Fabry Center (IFAZ), University Hospital Muenster, Germany) - 40 min
19:00-21:00
Banquet @ The Prince Park Tower Tokyo 33F
Day 2 / Oct. 3 (SAT)
9:00-10:20
- Chair
- Hiroshi Kobayashi (The Jikei University School of Medicine, Japan)
Toya Ohashi (The Jikei University School of Nursing, Japan
)
1) AAV Gene Therapy in Fabry disease
Robert J Hopkin (Cincinnati Children’s Hospital Medical Center, USA) - 40 min
2) Lentivirus-Mediated Gene Therapy for Fabry Disease: Long-Term Results and New Initiatives
Jeffrey A Medin (Medical College of Wisconsin, USA) - 40 min
10:20-12:50 (Co-sponsored by JCR)
- Chair
- Kimitoshi Nakamura (Kumamoto University Graduate School of Medical Sciences, Japan)
Yin-Hsiu Chien (National Taiwan University, Taiwan)
Newborn screening of Fabry disease: Workshop
Kimitoshi Nakamura / Takaaki Sawada (Kumamoto University Graduate School of Medical Sciences, Japan)
Yin-Hsiu Chien (National Taiwan University, Taiwan)
Dau-Ming Niu (National Yang Ming Chiao Tung University, Taiwan)
Barbara K Burton (Northwestern University, USA)
13:00-14:10 Luncheon seminar (Co-sponsored by Amicus Therapeutics)
- Chair
- Derralynn A. Hughes (University College London, UK)
Norio Sakai (ISEIKAI International General Hospital, Japan)
Pharmacogical Chaperones: New Frontier
■ Clinical response of migalastat for Fabry disease in Japan
Masahisa Kobayashi (The Jikei University School of Medicine, Japan) - 30 min
■ Alternative disease mechanisms and targets in Fabry disease
Derralynn Hughes (University College of London and Royal Free London NHS Foundation Trust, UK) - 40 min
14:10-14:20
Closing Remark
Masahisa Kobayashi (The Jikei University School of Medicine, Japan)