Program(Tentative)

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) Sudden ventricular tachycardia and fibrillation in male late-onset Fabry disease

Toraaki Okuyama (The Jikei University School of Medicine, Japan)

2) Reduced recurrence of cerebrovascular events following 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)

10:30-10:40

Coffee break

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 (Juntendo University, Japan)

Fabry disease: Real World evidence (RWE) of Treatment

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 Chairperson, Fujita Gakuen Educational Foundation, 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) TBA

Camilla Tøndel (Univ of Bergen, Norway)(Web) - 40 min

15:40-15:50

Coffee break

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) Multimodality imaging for the evaluation of cardiac involvement in Fabry disease

Kenichi Hongo (The Jikei University School of Medicine, Japan) - 30 min

2) TBA

Yoshihiko Nakazato (Saitama Medical University, Japan) - 30 min

3) TBA

Alessandro Burlina (University Hospital of Padova, 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: Effects, measurement challenges and future directions

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 Medicine, 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)

Alberto Burlina (University Hospital of Padova, Italy)

Discussion 10 minutes included

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

■ Japanese Experience

Masahisa Kobayashi (The Jikei University School of Medicine, Japan) - 30 min

■ New Frontier in Fabry Management

Derralynn Hughes (University College of London, UK) - 40 min

14:10-14:20

Closing Remark

Masahisa Kobayashi (The Jikei University School of Medicine, Japan)