Roche's study into a treatment for Angelman Syndrome has been approved by the medical ethics committee (CCMO and METC) and that means that now also Dutch children with Angelman syndrome can participate. The official name of the study is “Tangelo: an open-label, multicenter study to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of RO7248824 in participants with Angelman syndrome (BP41674)”.
In this study, participants are treated with an anti-sense oligonucleotide, which is a small piece of genetic material that turns on the UBE3A gene. This way brain cells can make the protein that children with Angelman syndrome lack.
In order to get the medicine in the right place, it must be given via an epidural. This is done under anesthesia. Several injections are required in the study, which lasts a year. Children with Angelman syndrome between the ages of 1 and 12 can participate. There are other criteria that participants must meet. If you would like more information, or if you would like to know whether your child or your patient could participate in the study, please contact us at firstname.lastname@example.org.