Azafaros A.g.
Clinical trials sponsored by Azafaros A.g., explained in plain language.
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New pill aims to slow rare brain diseases in children
Disease control Recruiting nowThis study tests an oral drug called nizubaglustat in children aged 4 and older with Niemann-Pick type C, GM1, or GM2 gangliosidosis. These are rare genetic diseases that damage the brain and body. The goal is to see if the drug can safely slow disease progression over 18 months.…
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 01, 2026 15:59 UTC
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Hope for kids with rare brain diseases: new pill trial launches
Disease control Recruiting nowThis study tests an oral drug called nizubaglustat (AZ-3102) in people aged 4 and older with GM1 or GM2 gangliosidosis—rare genetic disorders that damage the brain and nerves. Over 18 months, half the participants will receive the drug and half a placebo to see if it slows diseas…
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated Apr 30, 2026 15:52 UTC
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New pill could slow rare brain disease in kids – phase 3 trial underway
Disease control Recruiting nowThis study tests an oral drug called nizubaglustat in children and teens (ages 4 and up) with Niemann-Pick type C disease, a rare genetic disorder that affects movement and thinking. The trial lasts 18 months and compares the drug to a placebo to see if it can slow the disease an…
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated Apr 29, 2026 15:04 UTC