Azafaros for Patients
A beacon for people living with rare metabolic disorders
Azafaros is a company that seeks to develop new treatments to meet the needs of patients suffering from rare genetic metabolic disorders. We will do this by bringing together our deep understanding of these diseases and our extensive experience in developing new therapies, combined with the newest scientific insights.
We are proud to collaborate with Professor Hans Aerts, whose team led the discovery of AZ-3102, and the University of Leiden.
Equally important to our scientific approach is the collaboration with patients and patient organizations, to learn from their experience in living with these indications and where we as company can make a significant difference for them and their families.
Azafaros is inspired by patients and their families to start the journey toward disease-altering treatment options. In this section we aim to be a resource and provide the most up-to-date information on our progress as well as contribute to our ongoing dialogue with the patient community and the clinicians who treat them.
The Impact of Ataxia
Niemann-Pick UK
Annual Family Conference & Interactive Workshop 2022
In the following video, originally shared at the Annual Family Conference & Interactive Workshop 2022, Jodie Ann O’Grady describes the impact of Ataxia on her son Joshua and explains why there needs to be more awareness of this subject, particularly from clinicians. The video also features Professor Paul Gissen (UCL) who gives expert insight into how Ataxia can be one of the ways in which Niemann-Pick disease type C can be diagnosed.
Patient Resources

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