Glycosphingolipid lysosomal storage disorders
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.
- Niemann-Pick type C disease
- GM1 gangliosidosis
- GM2 gangliosidosis
The sponsor’s clinical activity is directed toward neurologic manifestations associated with these diseases, including movement impairment and progressive functional decline.
Niemann-Pick type C disease
Clinical research includes treatment evaluation in late-infantile and juvenile forms of NPC disease, with attention to disease-related neurological symptoms and patient function.
- Ataxia
- Juvenile-onset disease
- Late-infantile disease
Work in this area reflects a therapeutic interest in addressing central nervous system involvement in a rare metabolic disorder.
GM1 and GM2 gangliosidoses
The sponsor is also active in GM1 gangliosidosis and GM2 gangliosidosis, targeting the neurologic burden of these lysosomal storage diseases across paediatric and juvenile presentations.
- Late-infantile onset
- Juvenile onset
- Motor dysfunction
These studies align with therapeutic interest in disorders marked by progressive impairment of movement and neurological development.
Pharmacology and drug disposition
Azafaros B.V. also funds research into the absorption, metabolism, excretion, and bioavailability of nizubaglustat and AZ-3102, supporting its development in rare disease therapy.
- Metabolic profiling
- Pharmacokinetics
- Food effect
This area includes evaluation in healthy volunteers and healthy male subjects, with emphasis on understanding how the compound behaves in the body after oral administration.
