Below you will find a preliminary version of the programme. We will update this page when more definite information becomes available.

Besides the main conference programme as shown in the outline below, this conference will provide separate social programs for  adult patients and  adolescent patients and siblings. Child care (4 till 12 years of age) will be available during the conference. The content of the different programmes will also be shown below.

Thursday July 7th, 2022
Research meeting
(Closed session for medical professionals, researchers and CNE representatives)
12:00 12:30 Registration of participants research meeting
12:30 13:00 Walking lunch
Subject Speaker
A. Novel disease models for cystinosis
13:00 13:05 Welcome address> Jennifer Hollywood
13:05 13:25 In vitro models: Organoids => In vivo models Rat Model Jennifer Hollywood
13:25 13:40 MFSD12: a novel therapeutic target in cystinosis - Everolimus & cysteamine combination therapy Jennifer Hollywood
13:40 13:45 Q&A
13:45 14:05 In vitro models: Tubuloids Manoe Janssen
14:05 14:25 In vivo models: Rats Ester de Leo
14:25 14:35 Q&A
14:35 14:50 Tea break & poster session
B. Towards innovation in treatment of cystinosis
Novel treatment strategies
14:50 15:10 Gene-editing in cystinosis Manoe Janssen/Elena Sendino Garvi
15:10 15:30 mRNA therapy Tjessa Bondue
15:30 15:40 Q&A
Novel therapeutic targets & promising drugs
15:40 15:55 SC-ELOX Paul Goodyer
15:55 16:10 Novel mechanisms and therapy for kidney injury in nephropathic cystinosis Minnie Sarwal
16:10 16:20 Q&A
Innovation in treatment of ocular cystinosis
16:20 16:30 Novel eyedrop formulations for corneal complications in cystinosis Morgan Fedorchak
16:30 16:40 Biomaterial mediated transfer of CTNS for ocular cystinosis Valeria Graceffa
16:40 16:50 Development of contact lenses for the ocular complications of cystinosis Anuj Chauhan
16:50 17:00 Q&A
17:00 17:05 Final comments on research meeting Martine Besouw & Koenraad Veys
Thursday July 7th, 2022
Paramedical Meeting
(Closed session for paramedical professionals and CNE representatives)
12:00 12:30 Registration of participants paramedical meeting
12:30 13:00 Walking lunch
Round Table Discussion
13:00 17:00 How to fit the intake of cysteamine with the food & drinks schedule
Transitioning from tube to oral feeding
Therapy compliance
Transition from paediatric to adult care
Inputs from outside the organising committee
14:35 14:50 Tea break
Thursday July 7th, 2022
Main Conference
17:30 19:30 Welcome reception & registration Patient team
Friday July 8th, 2022
Main Conference
08:00 08:30 Registration and Welcome coffee Patient team
Subject Speaker
08:30 08:45 Opening Elena Levtchenko, Anne-Marie O’Dowd, Claudia Sproedt & Fons Sondag
08:45 09:15 Kick-off: What do you know about cystinosis? Interactive-quiz Patient team
Plenary session 1
Cystinosis: a global view - Session chair: Francesco Emma
Subject Speaker
09:15 09:45 Overview of cystinosis treatment in 2022 Elena Levtchenko
9:45 10:15 Availability of treatment for cystinosis patients around the globe Aurélia Bertholet Thomas
10:15 11:15 Bring good treatment for cystinosis patients around the globe (Pitches of 5 minutes & panel discussion) Moderator Rezan Topaloglu MD’s and patients
11:15 11:45 Coffee break All
Plenary session 2
How to monitor cysteamine treatment?- Session Chair: David Cassiman
11:45 12:00 Monitoring of cysteamine treatment laboratory perspective Leo Kluijtmans
12:00 12:30 Monitoring of cysteamine treatment patient perspective Marlies Cornelissen
12:30 12:45 Q&A Monitoring of cysteamine treatment Presenters
12:45 13:45 Lunch All
Plenary session 3
How to improve current treatment? - Session Chair: Koenraad Veys
13:45 14:15 Overview of cystinosis research Francesco Emma
14:15 14:45 New and improved medication for cystinosis: cysteamine prodrug Herbie Newell
14:45 15:15 Long-term follow-up of nephropathic cystinosis: importance of international registries Aude Servais
15:15 15:45 Medications after kidney transplantation and improved treatment of infections Paul Grimm
15:45 16:15 Tea break All
16:15 16:45 S4: Neurocognitive functioning in cystinosis patients John J. Foxe
16:45 17:15 Short poster presentations Prep. Koenraad Veys, Martine Besouw
17:15 18:00 Poster Session 1 All
Parallel sessions: social events
18:00 20:00 Dinner at own cost
19:00 22:00 Social Event Adult Patients: Cooking workshop Adult patients only
20:00 21:30 Walking tour Leuven All
Saturday July 9th, 2022
Main Conference
08:00 08:30 Welcome coffee
Subject Speaker
Plenary session 4:
Cystinosis as a multi organ disease - Session chair: Katharina Hohenfellner
08:30 09:00 Bone disease in cystinosis including surgery Dieter Haffner
09:00 09:30 Muscle disease in cystinosis Pascal Laforêt
09:30 09:45 How to improve muscle function in cystinosis: IMPACT stud Christian Koeppl
09:45 10:15 Eye complications in cystinosis & status of eye treatment Hong Liang
10:15 10:45 Panel discussion on use of eyedrops Ingele Casteels
10:45 11:15 Coffee break All
11:15 11:45 Pregnancy and cystinosis Graham Lipkin
11:45 12:15 Male fertility and cystinosis Koenraad Veys
12:15 13:45 Lunch Patients & Families
Parallel sessions:
09:00 12:15 Cartoon workshop Patients & Siblings age 13-18 only
13:45 14:45 Motivational speaker Joyce de Ruiter Patients & families
12:15 14:45 Consensus treatment goals in cystinosis (incl. lunch) MD’s & researchers Chairs: Martine Besouw & Koenraad Veys
Plenary session 5:
Future perspectives in cystinosis: From new born screening to gene therapy - Session chair: Martine Besouw
14:45 15:15 New-born screening for cystinosis & the effect of pre-symptomatic start of cysteamine treatment Katharina Hohenfellner
15:15 15:45 Status of autologous HSC transplantation in cystinosis & Condition of the patients Stephanie Cherqui
15:45 16:00 Stem cell transplant followed by living donor kidney transplant leaving the patient free of immunosuppression. Paul Grimm
16:00 16:30 Q&A gene therapy All
16:30 17:00 Tea break All
Parallel sessions:
17:00 18:00 Poster Session 2 All
17:00 17:30 Innovative tools for patients:
  • Patients' empowerment tool
  • Cystinosis & Me App
 Elena Levtchenko
Plenary sessions:
19:00 21:00 Conference dinner All
20:15 20:30 Best research poster & presentation awards Koenraad Veys, Martine Besouw, Marjolein Bos, Fons Sondag
Sunday July 10th, 2022
Patient & Family Workshops
(Closed session for patients & parents)
08:30 09:00 Welcome coffee
Subject Speaker
Plenary session 6:
Quality of life & Workshops - Chair: Elena Levtchenko
09:00 09:45 Results quality of life survey, Leuven Lore Willem
09:45 10:00 Introduction to the workshops Fons Sondag
10:00 10:30 Coffee break All
Parallel Sessions: Workshops
Parents do not (both) have to attend the workshop belonging to the age group of their child. Attending a higher age group gives information on what they can expect. Except for the youngest age group no medical doctors will participate in the workshops. The best way for adolescent patient to exchange information is probably not a formal workshop. Instead a Graffiti tour through Leuven is offered. Be aware of the earlier starting time. Siblings are welcome too!
10:30 12:00 Adult patient workshop:
How to cope with cystinosis: school, jobs, private life, friends, relations. What psycho-social support do patients need?		 Patient team
10:15 12:00 Adolescent patients & siblings 12-18:
Graffiti tour Leuven		 Patient team
10:30 12:00 Parents of adults:
Can we ever give up: parent adult child relation, loss of control, support autonomy, how to overcome setbacks		 Patient team
10:30 12:00 Parents adolescents 12-18:
What will the future bring: compliance, transfer to adult clinic, helping to accept the condition, cope with bullying, our own feelings and struggles as parents		 Patient team
10:30 12:00 Parents of young children 6-12:
Discuss day to day issues: taking medicines at school, get support at school, how to tell the other children in the group, playing and sleeping at a friend's home		 Patient team
10:30 12:00 Parents of young children 0-6:
When do we get rest from coping with: thirst, polyuria, vomiting, feeding problems/tubes, family support, paying attention to siblings		 Patient team, Elena Levtchenko
Plenary session 6: - Chair: Elena Levtchenko
12:00 12:15 Conference closure Summary of conference by photos & drawings Farewell Elena Levtchenko, Marjolein Bos, Fons Sondag
12:15 13:00 Lunch All