Niemann-Pick disease, type C
Clinical trials for Niemann-Pick disease, type C explained in plain language.
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New pill shows promise for rare brain disorders in early trial
Disease control CompletedThis study tested an oral drug called AZ-3102 in 13 people with GM2 gangliosidosis (Tay-Sachs or Sandhoff disease) or Niemann-Pick type C disease. The main goal was to check safety and how the drug moves through the body over 12 weeks. It was a randomized, double-blind, placebo-c…
Matched conditions: NIEMANN-PICK DISEASE, TYPE C
Phase 2 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC
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Promising drug tested for rare liver disease in newborns
Disease control CompletedThis early-phase study tested a drug called adrabetadex (VTS-270) given through a vein to treat liver disease in infants with Niemann-Pick disease type C, a rare and fatal genetic disorder. Four babies up to 6 months old received multiple doses to see if the drug could lower abno…
Matched conditions: NIEMANN-PICK DISEASE, TYPE C
Phase 1/2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 08:08 UTC