Webinars | Innovative Therapies for Machado-Joseph Disease and Other Hereditary Ataxias: From the Laboratory to the Patient

Background

Hereditary ataxias, such as Machado-Joseph Disease (SCA3), are rare neurodegenerative diseases that impair motor coordination, balance and other essential functions. For patients and their families, dealing with this condition means facing daily challenges arising from the progressive loss of mobility, which makes specialised support necessary over time. Despite scientific advances, the lack of accessible and reliable information is still a problem. Doubts about the evolution of the disease, the treatments available and the latest research can generate anxiety and insecurity. In addition, the lack of clear information also makes it difficult for patients to get involved in important decisions about their own health. To help bridge these gaps, we are proposing a series of educational seminars organised in collaboration with research institutions. These include the Centre for Neuroscience and Cell Biology and the Faculty of Science and Technology of the University of the Azores. In addition, there are patient organisations such as APAHE, AAADMJ and AMA Doentes Machado-Joseph. These online sessions will create an accessible and interactive space where experts will share important knowledge for understanding the disease, research and clinical follow-up. As well as clarifying doubts, the aim is to strengthen the network of collaboration between the various players, such as patients and their families, researchers, clinical bodies and the pharmaceutical industry.

Objectives

The main objectives of this cycle of seminars are:

- Strengthen scientific literacy in ataxias

- To publicise advances in translational research

- Prepare for participation in clinical trials

- Facilitate dialogue between patients, researchers and clinicians

This series of seminars aims to reduce the information gap between research and the ataxia patient community, promoting a more in-depth knowledge of the disease and the therapeutic strategies under development. By empowering patients with rigorous but understandable scientific information, we encourage more active and informed participation in research and clinical trials. Furthermore, by strengthening the interaction between patients, clinicians, the pharmaceutical industry and researchers, we contribute to a more collaborative research ecosystem aligned with the needs of the community.

How does it work?

The series of seminars will be held via synchronous sessions, broadcast in real time. These sessions allow participants to interact online with the speakers, clarify doubts and share curiosities.

To access the sessions, all you have to do is register in advance, ensuring quick and easy entry.

Each session will 45 minutes maximum, in which a guest will give a short 15-minute presentation, covering the topics clearly and objectively, followed by an interactive space for questions and answers.

The target audience will be patients and their families; however, the sessions will be open to the entire community.

It should be noted that the sessions will be recorded, but the interaction between the patients and the speakers will not. In addition, anyone taking part in the sessions will not be recognised, as their name will be hidden, thus guaranteeing the privacy of all participants.