| 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 |
| 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 |
