Enrollment Closed

Research into the use of the drug Gaboxadol in children with Angelman Syndrome completed

August 29, 2023Category: Editors Choice • Enrollment Closed

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

January 26, 2023Category: Participate in clinical studies • Enrollment Closed

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.

Tot nader bericht vol – Roche Tangelo studie open voor inclusie van kinderen in ENCORE Angelman syndroom expertisecentrum van Erasmus MC

December 23, 2020Category: Participate in clinical studies • Enrollment Closed

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.