NIEMANN-PICK TYPE C DISEASE
Clinical trials for NIEMANN-PICK TYPE C DISEASE explained in plain language.
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New hope for rare brain diseases: long-term safety trial of nizubaglustat begins
Disease control Recruiting nowThis study tests the long-term safety and effects of a daily drug called nizubaglustat in people with two rare genetic diseases that affect the brain: GM2 gangliosidosis and Niemann-Pick type C. About 21 participants who were in an earlier study or who are currently on another dr…
Matched conditions: NIEMANN-PICK TYPE C DISEASE
Phase: PHASE2 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 15, 2026 11:57 UTC
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New pill aims to slow rare brain diseases in kids
Disease control Recruiting nowThis study tests an oral drug called nizubaglustat in children aged 4 and older with Niemann-Pick type C disease or GM1/GM2 gangliosidosis. The goal is to see if the drug can safely slow the progression of these rare, serious brain disorders. Participants will take the drug or a …
Matched conditions: NIEMANN-PICK TYPE C DISEASE
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 13, 2026 15:59 UTC
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New pill could slow rare brain diseases in kids
Disease control Recruiting nowThis study tests an oral drug called nizubaglustat in children and teens aged 4 and older with Niemann-Pick type C disease or related disorders. The goal is to see if it can safely improve movement and coordination over 18 months. Participants are randomly assigned to receive the…
Matched conditions: NIEMANN-PICK TYPE C DISEASE
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 11, 2026 20:38 UTC