Study on the Effectiveness and Safety of Nizubaglustat in Patients with Niemann-Pick Type C, GM1, or GM2 Gangliosidosis
- Substances:NIZUBAGLUSTAT
France- Participants:0–17 years · 18–64 years
- Substances:NIZUBAGLUSTAT
- Sponsor:Azafaros B.V.
Rare diseases
Investigational molecules
Locations

J.H. Oortweg 21, 2333 CH Leiden, The Netherlands
Azafaros B.V. focuses on rare inherited disorders involving abnormal glycosphingolipid accumulation, with research centred on conditions that affect the nervous system and other organs.
The sponsor’s clinical activity is directed toward neurologic manifestations associated with these diseases, including movement impairment and progressive functional decline.
Clinical trials run by this sponsor – those enrolling participants first.
France
The NetherlandsIncrease visibility for your trials and help patients find your site through our platform.
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