Azafaros Media Release | July 16, 2024
- ANPDF
- Jul 17, 2024
- 1 min read
Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C
Read the full statement from Azafaros here:
Here is a summary of the article:
Positive Phase 2 Results: The RAINBOW study showed that nizubaglustat was safe and well tolerated in patients with GM2 gangliosidosis and Niemann-Pick disease type C.
Next Steps: Azafaros plans to start Phase 3 trials in 2025, with data to be presented at the SSIEM Annual Symposium in September 2024.
Nizubaglustat: This drug has a unique dual mode of action and is being developed for rare lysosomal storage disorders with neurological involvement.
Support and Designations: Nizubaglustat has received various designations from the FDA, EMA, and MHRA, highlighting its potential as a treatment for these rare diseases.
For more information, visit the company's website, azafaros.com
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