Azafaros A.g.
Clinical trials sponsored by Azafaros A.g., explained in plain language.
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New hope for rare childhood brain disease: major trial tests first oral treatment
Disease control Recruiting nowThis study is testing whether a daily oral medication called nizubaglustat can safely slow the progression of Niemann-Pick Type C (NPC), a rare and severe genetic disorder that affects the brain. It will involve about 72 children and young people (ages 4+) with NPC over 18 months…
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated Apr 02, 2026 02:41 UTC
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Race against time: new pill aims to slow devastating childhood brain disease
Disease control Recruiting nowThis study is testing whether a daily oral medicine called nizubaglustat can slow the progression of two rare, severe genetic brain diseases (GM1 and GM2 gangliosidosis) in children. For 18 months, about 75 participants aged 4 and older will receive either the real medicine or a …
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC
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New drug offers hope for families battling rare, devastating brain diseases
Disease control Recruiting nowThis study aims to gather long-term safety and effectiveness data for the drug nizubaglustat in people with rare, progressive brain disorders (GM2 gangliosidosis or Niemann-Pick type C). It includes two groups: patients continuing from a previous study and patients switching from…
Phase: PHASE2 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated Mar 27, 2026 12:39 UTC
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New hope for kids with devastating rare brain diseases
Disease control Recruiting nowThis study is testing whether an oral drug called nizubaglustat can safely slow down or control the progression of several rare, inherited brain diseases in children. It will involve about 147 participants, aged 4 and older, who have conditions like Niemann-Pick Type C or GM1/GM2…
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC