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 drug trial launches
Disease control Recruiting nowThis study tests the long-term safety and effects of a daily medication called nizubaglustat in people with two rare genetic brain diseases: GM2 gangliosidosis and Niemann-Pick type C. About 21 participants who were in a previous study or who are already on a similar drug will ta…
Matched conditions: NIEMANN-PICK TYPE C DISEASE
Phase: PHASE2 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 22, 2026 14:04 UTC
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New pill aims to slow rare childhood brain diseases
Disease control Recruiting nowThis study tests an oral drug called nizubaglustat in children and teens aged 4 and older with rare genetic brain diseases: Niemann-Pick type C, GM1, or GM2 gangliosidosis. The drug is taken by mouth for 18 months and compared to a placebo. The goal is to see if it can safely slo…
Matched conditions: NIEMANN-PICK TYPE C DISEASE
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 22, 2026 14:04 UTC
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New pill could slow rare brain disease in kids
Disease control Recruiting nowThis study tests an oral medication called nizubaglustat in children aged 4 and older 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 worsening of coor…
Matched conditions: NIEMANN-PICK TYPE C DISEASE
Phase: PHASE3 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 22, 2026 13:50 UTC