ONLINE   FAMILY   CONFERENCE

Saturday 25th April 2020

Start 13:00 CEST

Please check your timezone

Keynote Speaker

Dr Stephanie Cherqui, University of California, San Diego

Session 1: Cystinosis and its management

13:00 – 14:00 (CEST)

Welcome & Introductions

13:00 – 13:10

Anne Marie O’Dowd

Chair of Cystinosis Network Europe and Global Cystinosis Community Advisory Board

Cystinosis and its management

13:10 – 13:30

Prof Elena Levtchenko – Conference Scientific Chair

Head of Department of Paediatric Nephrology, University Hospitals Leuven Belgium

Cystagon and Procysbi – How they work, why they are important and potential new treatments

13:30 – 13:50

Prof Donald Cairns

Head of School of Pharmacy and Life Sciences, Robert Gordon University Aberdeen

13:50 – 14:00 Break

Session 2

14:00 – 15:00 (CEST)

Working with schools – a shared language for cystinsois

14:00 – 14:20

Dr Joyce Senior

Educational Psychologist; Asst Professor of Education and Director, Professional Doctorate in Educational Psychology, University College Dublin

Maintaining your Mental Health

14:25 – 14:40

Dr Siobhán MacHale

Consultant Liaison Psychiatrist, Dept of PsychiatryBeaumont Hospital, Senior Lecturer RCSI, Dublin

14:45 – 15:00 Break

Session 3: The Eyes

15:00 – 16:00 (CEST)

The Eyes in Cystinosis

15:00 – 15:20

Rachel Bishop, MD, MPH

Staff Ophthalmologist (contractor), National Eye Institute, National Institutes of Health, Bethesda, Maryland, U.S.A.

A possible new method of delivering cystamine to the eyes in cystinosis

15:25 – 15:45

Prof Anuj Chauhan

Department Head of Chemical and Biological Engineering, Colorado School of Mines

15:50 – 16:00 Break

Session 4: Muscles and Bones

16:00 – 17:00 (CEST)

Muscle and Bone issues – treatments and latest research

16:00 – 16:20

Professor Justine Bacchetta MD, PhD

Professor of Pediatrics, CHU de Lyon HCL – GH Est-Hôpital Femme Mère Enfant, Lyon, France

Specialist in pediatric nephrology and pediatric diseases of calcium and phosphate metabolism

Benefit of exercise, exercises to do at home & new research on Galileo training boards for cystinosis

16:25 – 16:45

Christian Köppl

Physiotherapist at the Centre of Social Paediatrics in Traunstein, Germany

16:50 – 17:00 Break

Session 5

17:00 – 18:00 (CEST)

Kidney Transplant: Planning, Procuring and Preservation

17:00 – 17:30

Prof Paul Grimm

Professor of Pediatrics; Medical Director, Pediatric Kidney Transplant Program; Dept of Pediatric Nephrology Lucile Packard Children’s Hospital; Stanford University School of Medicine

Pregnancy for Women with Cystinosis

17:30 – 17:45

Dr Aude Servais

Adult Nephrologist, Necker Hospital, Paris

Male Fertility in Cystinosis

17:45 – 18:00

Dr Ahmed Reda

KU Leuven, Belgium

18:00 – 18:15 Break

Session 6: Latest research in cystinosis

18:15 – 19:45 (CEST)

Overview of the Latest Research

18:15 – 18:30

Dr Patrick Harrison

Department of Physiology, University College Cork

Keynote Address:

Clinical Trial for Autologous Transplantation of Gene-Corrected Hematopoietic Stem Cells for Cystinosis

18:30 – 19:00

Dr Stephanie Cherqui

Associate Professor in Department of Pediatrics, Division of Genetics, University of California San Diego

The Discovery and Development of CF10 – a cysteamine prodrug to improve the lives of those with cystinosis

19:00 – 19:15

Prof Herbie Newell

Professor of Drug Development at the University of Sunderland and Emeritus Professor of Cancer Therapeutics at Newcastle University, England

A No Nonsense Approach to Cystinosis: a new treatment potential

19:15 – 19:30

Prof Paul Goodyer

Professor of Pediatrics and Human Genetics, McGill University Health Centre, Montreal

A Novel Urine Test to Track Kidney Injury and Transplant Rejection

19:30 – 19:45

Prof Minnie Sarwal

Professor in Residence, Surgery, Medicine, Pediatrics, UC San Francisco (UCSF); Medical Director, Kidney Pancreas Transplant Program, Director, Precision Transplant Medicine

19:45 – 20:00 Break

Session 7:  The Expert Panel

Open Q & A with our speakers from today and joined by Dr Katharina Hohenfellner, Germany and Dr Atif Awan, Dublin

20:00 – 21:00 (CEST)

Cystinosis Ireland is the proud host of the Virtual Cystinosis Conference 2020. Without funding and donations this conference could never take place.
We would like to thank all our supporters.