Cara Gilmore video (the impact of dysarthria in Niemann-Pick)
With the support of Azafaros, the Niemann Pick UK charity created this impactful video, in which Cara Gilmore shares her story about the impact of speech problems in Niemann-Pick Type C.
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.
AZ-3102 Mode of Action Treatment development for GM1 and GM2 gangliosidosis
This video has been created by Azafaros in close collaboration with the GM1 and GM2 community to provide information on how a potential treatment developed for GM1 and GM2 gangliosidosis may work. It aims to explain the diseases and educate patients and families on the possible mechanism and mode of action of Azafaros’ compound, AZ-3102.
To select a suitable language, click the orange button to go to the original movie. There you can choose from English, German, Brazilian Portuguese or Spanish (Latin America).
What is the PRONTO study?
PRONTO is a natural history study aiming to understand how GM1 and GM2 gangliosidoses progress over time, which might help researchers understand how the condition develops and possibly how to treat it in the future. In the short video below, you can learn more about what PRONTO is, and why it is important.