| 08.30 - 09.00 |
Envisioning the development of therapautic modalities - Nicole Wolf |
| 09.00 - 09.30 |
Envisioning the future of gene therapy - Niek van Til |
| 09.30 - 10.00 |
Molecular targets for therapy - Truus Abbink |
| 10.00 - 10.30 |
Clinical outcome measures - Marc Engelen |
| 10.30 - 11.00 |
Tea and coffee |
| 11.00 - 12.30 |
Free presentations part II |
| |
- Hamilton: Macrophage transplantation rescues RNASET2-deficient leukodystrophy by replacing deficient microglia in a zebrafish model
15 minutes
- Gritti: Exploring myeloid-mediated enzymatic correction of ARSA-deficient neural cells in hematopoietic stem cell gene therapy for Metachromatic Leukodystrophy
15 minutes
- Pujol: Novel biomarkers and therapeutic hope for DEGS1 leukodystrophy
15 minutes
- Videbæk: C26:0-lyso-phosphatidylcholine Levels at Birth Predict Phenotype in X-Linked Adrenoleukodystrophy: A Multicenter Retrospective Cohort Study 10 minutes
- Schäfer: Improving quality of life in symptomatic women with X-linked adrenoleukodystrophy by a web-based, multidisciplinary lifestyle intervention: A randomized-controlled clinical trial (SMART-ALD study)
10 minutes
- Grol: Cognitive and Intellectual Functioning in Vanishing White Matter and Metachromatic Leukodystrophy Patients: A Prospective Study
15 minutes
- van Voorst: Impact of VWM on family members
10 minutes
|
| 12.30 - 13.30 |
Lunch |
| 13.30 - 13.50 |
Registries for leukodystrophies and trial readiness - Daphne Schoenmakers |
| 13.50 - 14.10 |
The interface between medical researchers and industry - Prisca Leferink |
| 14.10 - 14.30 |
Prices for therapies - Carla Hollak |
| 14.30 - 14.50 |
Leukodystrophies: a field of collaboration - Ali Fatemi |
| 14.50 - 15.00 |
Closing - Nicole Wolf |
| |
|
| Location |
Vrije Universiteit (VU) - Aula |
| |
|
| 15.45 - 16.30 |
Farewell lecture (in Dutch) - Marjo van der Knaap |
| |
Reception |
