The Spanish Association of Orphan and Ultra Orphan Drugs (AELMHU) has announced the celebration of the third edition of the National Conference: The Future of Advanced Therapies for Rare Diseases. This event, for the third consecutive year, will once again be supported by several leading companies in the pharmaceutical sector, all of them members of the Association, such as AMGEN, CSL Behring, PTC Therapeutics, Ultragenyx and Vertex, with the technical secretariat of BioInnova Consulting.
Event details:
- Date: Tuesday, March 25, 2025
- Time: 9:30 a.m.
- On-site location: Espacio Bertelsmann (Calle de O'Donnell, 10, Madrid). Limited places.
- Online broadcast: The link to access will be provided in the days prior to the event, through the AELMHU YouTube channel.
- Registration: Interested parties can register through the form available here.
- Agenda: A real-time update of the participants' agenda is available at this link.
Objective of the day
The main purpose of this meeting is to promote the exchange of ideas and generate a deep reflection among the different actors in the healthcare field, in order to highlight the importance of Advanced Therapies in the treatment of rare diseases. In addition, it will seek to create a space for collaboration to ensure that these innovative treatments are accessible and available to patients.
Throughout the day, renowned experts will share their knowledge and experience on various aspects of advanced therapies. Some of the prominent topics that will be discussed include:
During this event, three key topics will be discussed to advance the diagnosis and treatment of rare diseases:
- Joint Clinical Evaluations: Keys to Innovation and Access to Advanced Therapies
A space to explore how joint clinical evaluations can improve innovation and facilitate access to advanced therapies, optimizing regulatory processes and accelerating the availability of treatments. - Diagnosing Rare Diseases: Neonatal Screening, Precision Medicine and Data Management
We will discuss the latest innovations in early diagnosis of rare diseases through neonatal screening, the move towards precision medicine and the importance of proper data management to further improve care and treatment. - Legislative Advances and Challenges in Addressing and Funding Advanced Therapies for Rare Diseases
We will discuss current legislative challenges and new initiatives that seek to ensure funding and access to advanced therapies for rare disease patients.
