The ROSA study for children with Angelman syndrome started in June 2021. In this study we are looking for suitable and feasible outcome measures for children with Angelman syndrome, and we want to learn more about the natural history of this rare syndrome.
Roche Tangelo study open for inclusion of children in ENCORE Angelman syndrome expertise center at Erasmus MC
Roche's study into a treatment for Angelman Syndrome has been approved by the medical ethics committee (CCMO and METC), which means that Dutch children with Angelman syndrome can now also participate.
In the TRAIN-study, we will conduct a study with trametinib in adult NF1 patients with plexiform neurofibromas that cause complaints.
Afgeronde klinische studies (deelname gesloten)
Phase 3 clinical study of the effect of the drug OV101 (Gaboxadol) in children with Angelman Syndrome
Phase 2 and 3 clinical study of the effect of Lamotrigine on cognition in patients with NF1
Cognitive variability in NF1 and TSC monozygotic twins
Phase 3 clinical study of the effect of Cannabidiol on epilepsy in patients with TSC
Phase 2 and 3 clinical study of the effect of the drug Sirolimus in children with TSC.
Fase 2 en 3 klinische studie naar het effect van het medicijn Sirolimus in kinderen met TSC.
Phase 3 clinical study of the effect of the drug Simvastatin in children with NF1
Phase 3 clinical study of the effect of the drug Simvastatin in patients with NF1