NIEMANN-PICK DISEASE, TYPE C
Clinical trials for NIEMANN-PICK DISEASE, TYPE C explained in plain language.
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Experimental drug targets liver disease in rare, fatal infant disorder
Disease control CompletedThis early-phase study tested a drug called adrabetadex in 4 infants with Niemann-Pick disease type C (NPC), a rare and fatal genetic disorder. The drug was given through a vein to see if it could help treat liver problems that often occur in newborns with NPC. The study measured…
Matched conditions: NIEMANN-PICK DISEASE, TYPE C
Phase: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated May 26, 2026 05:10 UTC
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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…
Matched conditions: NIEMANN-PICK DISEASE, TYPE C
Phase: PHASE2 • Sponsor: Azafaros A.G. • Aim: Disease control
Last updated May 26, 2026 04:37 UTC