Honeycomb study for GRIN patients started!
In September 2023, the GRIN expertise center started the Honeycomb study of GRIN Therapeutics.
Continue ReadingResearch into the use of the drug Gaboxadol in children with Angelman Syndrome completed
In 2019 and 2020, we participated in an international study with the ENCORE expertise center for Angelman Syndrome into the effect of the drug gaboxadol in children with Angelman Syndrome. This is a drug that stimulates the GABA delta receptor. Dutch children also participated.
In a previous study in which all participants received gaboxadol, clear positive effects were measured on daily functioning. In the current study, a lottery was done between gabaxadol and placebo. The results showed an improvement in both the children who received placebo and those who received gabaxadol. So there was no improvement with the gabaxadol. There were also no serious side effects. This again shows that a study with a placebo group is really important to know for sure whether a treatment is effective.
We thank the participants and their parents. The article with all the results has now been published. Click here to read the article (link valid until October 15)
ZEN study
Met de ZEN studie proberen we te onderzoeken of hoge resolutie zenuwechografie gebruikt kan worden voor het onderzoeken van de zenuwen van mensen met neurofibromatose type 1. Ook onderzoeken we of we veranderingen over de tijd kunnen meten.
Continue ReadingTot nader bericht vol – Roche Tangelo studie open voor inclusie van kinderen in ENCORE Angelman syndroom expertisecentrum van 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.
Continue ReadingNEPTUNE TRIAL
Phase 3 clinical study of the effect of the drug OV101 (Gaboxadol) in children with Angelman Syndrome
Continue ReadingEXCEL TRIAL
Phase 2 and 3 clinical study of the effect of Lamotrigine on cognition in patients with NF1
Continue ReadingCOVANT STUDY
Cognitive variability in NF1 and TSC monozygotic twins
Continue ReadingGWPCARE6 TRIAL
Phase 3 clinical study of the effect of Cannabidiol on epilepsy in patients with TSC
Continue ReadingRAPIT TRIAL
Phase 2 and 3 clinical study of the effect of the drug Sirolimus in children with TSC.
Continue ReadingRATE TRIAL
Fase 2 en 3 klinische studie naar het effect van het medicijn Sirolimus in kinderen met TSC.
Continue Reading