The 6th International Forum of Lysosomal Disorders
Program (March 30-April 1, 2023、Tokyo, Japan)
March ３０ （Thu） Day1

Open Memorial Lecture on Top Medical Science
First Floor, The Auditorium, 2nd building The Jikei University
16:00 – 16:20

Welcome Lecture
Prof. Yoshikatsu Eto (President, the 6th International LSD Forum)

16:20-16:30

Welcome Greeting
Prof. Senya Matsufuji (President, Jikei University, Tokyo)

16:30-17:00

Special Lecture I
Chair: Prof. Yoshikatsu Eto (Sothern Tohoku Institute for Neuroscience/JikeiUniversity, Tokyo)
Genetics, Genomics & Omics: Impact on Genetic Diseases"
Prof. Robert Desnick ( Mount Sinai Medical Center, N.Y.)

17:00-17:30

Special Lecture II
Chair: Prof. Kimihiko. Oishi (Jikei University, Tokyo)
Advances in Gene Therapy for Lysosomal Diseases – Trials and Tribulations
Prof. Brian Bigger (Manchester University)

17:30 – 18:10

Special Lecture III

Chair: Prof. Torayuki Okuyama (National Institute for Child Health and Development, Tokyo)

The Golden Age of Rare Disease Treatment
Dr. Emil Kakkis ( CEO and President, Ultragenyx, USA)
18:10 – 18:40

Special Lecture IV
Chair: Prof. Toya Ohashi (Jikei University, Tokyo)
Life Science Development in Japan
Prof. Ryuichi Morishita ( Osaka University)

19:00– 21:00

Welcome Party at the Prince Park Tower Tokyo, the 33th floor

March 31（Fri） Day 2
The 6th International Forum of Lysosomal Storage Diseases PART II
Tokyo Prince Hotel-Magnolia Hall
8:30 – 8:40

Opening Remarks
Prof. Yoshikatsu Eto (President, the 6th International LSD Forum)---------------------------------------------------------------------------------------------------------------------------------Workshop 1

Fabry disease
Chair: Prof. Robert J. Desnick(Mt Sinai Medical Center, NY) / Prof. Yoshikatsu Eto(Sothern Tohoku Institute for Neuroscience/Jikei University, Tokyo)

８:40 – 9:00
Chaperon therapy of Japanese Fabry patientsDr. Masahisa Kobayashi (Jikei University, Tokyo)
9:00 – 9:20

Clinical Phenotype and Genotype of Japanese Female Fabry Patients.
Dr. Kazuya Tsuboi (Nagoya Central Hospital, Nagoya)

Invited Lecture 1

Chair: Prof. Yoshikatsu Eto(Sothern Tohoku Institute for Neuroscience/Jikei University, Tokyo)
9:20 – 9:50

Fabry Disease: History, Subtypes and Treatment
Prof. Robert J. Desnick (Mt. Sinai Medical Center, N.Y.)

9::50-10:00

Break

Invited Lecture

2

I Invited

Chair: Prof. Hitoshi Sakuraba (Meiji Pharmaceutical University, Tokyo)

10:00 – 10:40

Biomarkers of Fabry disease
Prof. Johannes MFG Aerts ( University of Leiden, Netherlands)

Invited Lecture 3
10:40 – 11:20

Chair: Dr. Ken Sakurai (Jikei University, Tokyo)
Management of Fabry disease (Tentative)
Prof. Robert J. Hopkin（Cincinnati Children Hospital, US）

Invited Lecture 4
11:20-12:00

Chair: Prof. Kenichi Hongo（Jikei University, Tokyo）

Measuring cardiac involvement in Fabry disease and the use of AI
Prof. James Moon ( Barts Heart Centre (BHC), UCL, UK)Luncheon Seminar
Chair：Prof. Tsutomu Takahashi (Akita University, Akita)
12:10– 13:10■ Japanese cases of LAL-D

Dr. Yuki Yamada (Osaka City General Hospital)

Invited Lecture 5■ Lysosomal Acid Lipase: Deficiency diseases, and roles in cholesterol
metabolism and immune function
Prof. Gregory Grabowski (Cincinnati Children Hospital, USA)
Invited Lecture 6

13:20-14:00
Chair: Prof. N. Sakai (Osaka University, Osaka)
Next-generation proteomics to dissect Fabry disease plasticity

Prof. Soumeya Bekri（CHU, France）------------------------------------------------------------------------------------------------------------------------------------Workshop 2 Gaucher Disease
Invited Lecture 7
Chair: Prof. Kimitoshi Nakamura (Kumamoto University, Kumamoto)
14:00 – 14:30

Understanding of molecular and pathophysiology of GD3 patient with massive

Lymphadenopathy

Prof. Han-Wook Yoo (Asan Medical Center, Korea)
Chair: Prof. Pramod Mistry (Yale University, New Haven)/
Dr. Kei Murayama(Chiba Children Hospital, Chiba)
14:30 – 14:50

Clinical Phenotypes of Japanese Gaucher Disease
Dr. Aya Narita （Tottori University, Tottori）

14:50 – 15:10

SRT treatment in Japanese Gaucher disease
Dr. Ken Sakurai (Jikei University, Tokyo)

15:10-15:40
Chair: Dr. Kei Murayama(Chiba Children Hospital, Chiba)
Education Lecture 1

Inflammation mechanisms of Gaucher disease, biomarkers and therapeutic targets.
Prof. Pramod Mistry (Yale University, New Haven, US)
15:40-15:50

Coffee break――――――――――――――――――――――――――――――――――――――――――

Workshop 3: Mucopolysaccharidosis
Chair: Prof. Robert Giugliani (Federal University of Rio Grande do Sul, Brazil)
Dr. Motomichi Kosuga (national Institute for Child Health and Development, Tokyo)

Education Lecture 2
Chair: Dr. Motomichi Kosuga (national Institute for Child Health and Development,Tokyo)
15:50 – 16:20

MPS: overview
Prof. Roberto Giugliani (Federal University of Rio Grande do Sul, Brazil)

16:20 – 16:40

Japanese MPS II Mutations and Phenotype of MPS
Dr. Motomichi Kosuga (National Center for Child Heal and Development,Tokyo)

16:40 – 17:00

MPS VII and Nobel ERT
Prof. Takashi Hamazaki (Osaka Metropolitan University, Osaka)

17:00-17:10

Discussion

Workshop 4:

Novel Treatment for LSD

Invited Lecture 8
Chair: Prof. Toshiyuki Seto (Osaka Metropolitan University,Osaka)
17:10– 17:50

Treatments for MPS: Past, Present and Future”
Prof. Paul Harmatz (UCSF, US)------------------------------------------------------------------------------------------------17:50 – 18::10

Nobel Enzyme Treatment of Pompe disease
Dr. Keiko Ishigaki (Tokyo Womens Medical University, Tokyo)

Invited Lecture 9
Chair: Prof. Toya Ohashi ((Jikei University, Tokyo)
18:10– 18:50

Chaperon treatment of Fabry disease
Prof. Derralynn Hughes ( Royal Free Hospital London, UCL, UK)

19:00 –21:00- Banquet: Tokyo Prince Hotel (Sun Flower room)-April 1 (Sat) Day 3
The 6th International Forum of Lysosomal Storage Diseases PART III
Tokyo Prince Hotel-Magnolia Hall
8:10-8-40

Educatition Lecture 3
Chair: Prof. Yoshikatsu Eto (Sothern Tohoku Institute for Neuroscience/Jikei University,Tokyo)
Basic Pathology in Autophagy and Lysosomal Diseases
Prof. Norio Sakai (Osaka University, Osaka)Workshop 5: CNS Treatment
8:40-9:10

Education Lecture 4
Chair. Prof. Takashi Hamazaki （Osaka Metropolitan University, Osaka）
CNS treatment of MPS II
Prof. Torayuki Okuyama (Saitama University, Saitama)
9:10 –9:40

Invited Lecture 10
Chair : Prof. Hiroshi Kobayashi (Jikei University, Tokyo)
CNS treatment of MPS I, II
Prof. Roberto Giugliani (Federal University of Rio Grande do Sul, Brazil)
9:40 – 10:10

Education Lecture 5
Chair: Prof. Hitoshi Osaka (Jichi University School of Medicine, Tochigi)
CNS treatment of CLN2
Dr. Kaoru Eto (Tokyo Womens Medical University, Tokyo)

Workshop 6: Gene Therapy for LSD
Chair: Prof. Brian Bigger (Manchester University, UK)/
Prof. Masafumi Onodera (National Center for Child Health and Development,Tokyo)
10:10-10:30

Current Status of Gene Therapy in Japan
Prof. Masafumi Onodera (National Center for Child Health and Development, Tokyo)

10:30 –11:00

AAV gene therapy for Niemann Pick C1 and other diseases.
Prof. Takanori Yamagata (Jichi University, Tochigi)

11:00 –11:20

Gene Therapy for MPS II
Prof. Hiroshi Kobayashi (Jikei University, Tokyo)

Invited Lecture 11
Chair : Prof. Takanori Yamagata (Jichi University, Tochigi)
11:20 –11:50

Stem Cell Gene Therapy for Childhood Dementias – A Sanfilippo Stor0079
Prof. Brian Bigger (Manchester University, UK)

Luncheon Seminar
12:00 –12:40

Invited Lecture 12
Chair: Prof. Hiroyuki Ida（Jikei University, Tokyo）

A decade of eliglustat SRT transforming care of Gaucher disease
Prof. Pramod Mistry (Yale University, New Haven, US)――――――――――――――――――――――――――――――――――――――――――

Workshop 7: Newborn Screening of LSD
Invited Lecture 13
Chair: Prof. Kimihiko Oishi (Jikei University, Tokyo)
12:50 –13:30

Newborn screening of LSD In US

Dr. Jamie Mills (North Carolina US)

Invited Lecture 14:
Newborn screening in Asia
Chair: Prof. Yin-Hsiu Chien (National Taiwan University, Taiwan)/Prof. Shinichi Hirose (Fukuoka University, Fukuoka)
13:30- 14:00

Current situation in Taiwan
Prof. Yin-Hsiu Chien (National Taiwan University, ,Taiwan)

14:00 – 14:30

Current situation in Japan
Prof. Kimitoshi Nakamura (Kumamoto University, Kumamoto)

14:30 –14:40

Closing Remarks
Prof. Yoshikatsu Eto (President, the 6th International LSD Forum)

Contact to:■Research Center for Advanced Medical Science and Genetic Disease Treatment Research Institute,
Southern Tohoku Institute for Neuroscience , 255 Furusawa-Miyako, Asao-ku, Kawasaki, Kanagawa,
215-0026, Japan Tel: 044-322-0654 (3229 after electronic tone) Fax: 044-966-0133 E-mail: Sato<s-mariko@sato-sei.com>■Intergroup, 2-2-5 Toranomon, Minato-ku, Tokyo 105-0001, Japan Kyodo News Hall
forum-secret@intergroup.co.jp Contact: Miss Fujita