Azafaros A.g.
Clinical trials sponsored by Azafaros A.g., explained in plain language.
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New pill shows promise for rare genetic brain disorders
Disease control CompletedThis study tested an experimental oral drug, AZ-3102, in 13 people aged 12 and older with GM2 gangliosidosis (Tay-Sachs or Sandhoff disease) or Niemann-Pick type C disease. The main goals were to check the drug's safety and how it moves through the body over 12 weeks. Participant…
Phase: PHASE2 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 26, 2026 07:27 UTC
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New study tracks rare brain diseases in children
Knowledge-focused CompletedThis study followed 31 children with GM1 or GM2 gangliosidosis (including Tay-Sachs and Sandhoff disease) to see how their movement and speech changed over time. Researchers measured walking and talking abilities using a standard rating scale. The goal was to better understand th…
Sponsor: Azafaros A.G. • Aim: Knowledge-focused
Last updated May 26, 2026 07:42 UTC