CTNNB1 Foundtaion
With the team of prof. dr. Jerala we have been collaborating for the last two years, both in the development of potential gene therapy and their efforts to establish a CTGCT center in Slovenia. Such a center would mean exceptional progress in gene therapies in Slovenia and worldwide and give parents back the hope that there is a better future for their children. Moreover, in rare diseases such as CTNNB1, patients’ organizations must be full partners with clinicians and researchers for rapid and effective advancement of research. Therefore, the foundation supports the efforts for the project, and at the same time, we are committed to being active stakeholders in their activities upon successful establishment.
Špela Miroševič, MS Co-Founder, President
Junaki 3.Nadstropja
‘The 3rd floor Heroes’, founded in 2020, is a patient society that brings together parents of children with cancer. ‘The 3rd floor Heroes’ are children who are or have been treated at the haemato-oncology ward on the 3rd floor of the Paediatric Clinic in Ljubljana. The objectives of our society are financial, social and psychological assistance to heroes and their families during and after the treatment of this insidious disease, on the basis of their own experience. Within a short period of the society’s activity, we helped to improve the conditions for the treatment of children with cancer in the hemato-oncology department of the Paediatric Clinic in Ljubljana by amending medical legislation, numerous donations of medical and technical devices, renovation of premises and additional activities that increase the quality of hospitalization. Many children need tailored treatment, special diagnostic tests, and medical care abroad. The establishment of the Centre for Technologies of Gene and Cell Therapy (CTGCT) shows that the percentage of these will be lower and the percentage of cured will be higher.
Urška Kolenc, President of the 3rd floor Heroes society
IDefine Europe
IDefine Europe, Foundation for the Advanced Treatment of Rare Genetic Diseases is a non-profit organisation based in Slovenia and a member of Eurordis. It was established to connect different fields of science and healthcare for rare disease research. IDefine Europe is a partner organisation of IDefine (non-profit based in USA), together this two patient advocacy organisations have a global reach in advocating Kleefstra syndrome and are connecting researchers, clinicians and parents from across the globe. We want to improve the lives of children with neurodevelopmental disorders caused by rare genetic diseases. Based on data collection, data analysis and the generation of new knowledge, the primary purpose is to transfer knowledge, both to parents of children with rare diseases and to professionals. Focused on technology (AI/ML, data science), data, and knowledge transfer, we are trying to find new treatments and cures based on cell and gene therapies. We express our support for the establishment of the Centre for the Technologies of Gene and Cell Therapy. We believe that this Centre is very much needed and can serve to Europeans and beyond.
Tanja Zdolšek Draksler, PhD, founder and president of IDefine Europe, Foundation for the Advanced Treatment of Rare Genetic Diseases