Dr.
Danijela Szili

Persönliche Angaben:
Position / Funktion: President
Institution / Klinik / Universität: Hungarian Rett Syndrome Foundation
Land: Ungarn

Wissenschaftlicher Hintergrund:
Fachgebiet: Rett Syndrome, Translational research
Mitgliedschaften / Fachgesellschaften: EURORDIS DITA Task Force and RSE SAB

Spezialgebiete

• Patient advocacy
• Clinical trials

Vortrag beim Rett Summit 2027:
Titel: Rett Syndrome in Hungary
Sprache: English

Biografie:
Danijela Szili is a pharmacist, patient advocate, and recognized expert in Rett syndrome, with more than 15 years of experience working at the intersection of rare disease advocacy, translational research, medicines development, and patient-centred care. Since 2011, she has been actively involved as a parent and advocate for people living with Rett syndrome and their families, bringing a unique combination of professional pharmaceutical expertise, lived experience, multilingual communication skills, and a strong commitment to advancing rare disease research and access to innovative treatments.

Danijela has served as Chairwoman of the Hungarian Rett Syndrome Foundation since 2011, where she has worked to strengthen patient and family support, raise awareness of Rett syndrome, and promote collaboration between patients, clinicians, researchers, pharmaceutical companies, and regulatory stakeholders. Her extensive advocacy experience has enabled her to represent the perspectives and needs of the Rett community within national and European initiatives. At European level, Danijela has played an active role in Rett Syndrome Europe (RSE), including nine years as a member of its Board. She currently serves on the RSE Scientific Advisory Board and has contributed significantly to the organisation's efforts to strengthen research engagement. In 2019, on her initiative, RSE established a Research Subcommittee to increase the organisation's involvement in research. This initiative subsequently evolved into the RSE Scientific Advisory Board, providing a structured mechanism for integrating scientific expertise and patient perspectives into the organisation's work. Danijela's involvement extends beyond patient advocacy into medicines development and regulatory affairs. As a member of the EURORDIS DITA Task Force, she received training and gained practical experience in medicinal product development, regulatory processes, and access to medicines in Europe. She is also a registered Rett syndrome expert within the European Medicines Agency (EMA) network of European experts. Her regulatory experience includes participation in the EMA protocol assistance procedure for the Sarizotan HCl clinical trial in 2015, including participation in the Scientific Advice Working Party (SAWP) meeting concerning the product. She has contributed to several European and international initiatives related to Rett syndrome, including the EU Rett Delphi Expert Panel Project and the EU Rett Syndrome Policy Forum in Brussels. She has also served as a Rett syndrome patient and parent expert in consultancy work for Acadia Pharmaceuticals and as a member of the clinical development advisory board for fenfluramine.

Through these roles, Danijela has developed a strong understanding of the challenges involved in translating scientific research into meaningful outcomes for patients and families. Her work reflects a practical and goal-oriented approach to ensuring that patient perspectives are considered throughout the research and medicines-development pathway. With her background as a pharmacist, long-standing experience as a parent and advocate, and extensive involvement in European rare disease networks, Danijela is particularly well positioned to bridge the perspectives of science, patients, industry, and regulatory stakeholders. She is a highly skilled communicator who brings motivation, practical expertise, and a strong commitment to collaboration.