About this Talk
In this talk, Daniela Tropea presents the importance of clinical registries and databases for sharing data on Rett syndrome. In the last decades much progress has been made in understanding the biology of Rett Syndrome (RTT), and now some candidate treatments have reached advanced phases of clinical trials. Still, many candidate treatments have been discarded because of the variability of the symptoms in patients with Rett syndrome. We are missing the opportunity to identify treatments that improve the conditions of some (but not most) patients. Research in other disorders, such as autism and schizophrenia, shows that joining forces to increase the data available for analysis accelerates the research and leads to improved therapies. This is particularly valuable for Rett syndrome, where the variability of the presentation, paired with the rarity of the condition is hampering the progress in prognosis, patients’ management, and treatment.
The majority of the studies in Rett syndrome are based on a limited number of patients and do not allow a proper power for the analysis of the data. We need to increase the number of patients involved in research studies and to create a strategy that will facilitate the collection, access and sharing of data. To this aim Daniela and her team have designed a data platform: DATARETT, which can serve as basis for sharing data between clinicians, scientists and parents’ association.