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: PHASE2 • 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: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 08:08 UTC
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Scientists dive into rare cholesterol disorders to uncover clues
Knowledge-focused CompletedThis study looks at rare genetic disorders where the body can't make cholesterol properly, which can cause birth defects and learning problems. Researchers collect blood, urine, and tissue samples from affected people and their families to learn more about these conditions. The g…
Matched conditions: NIEMANN-PICK DISEASE, TYPE C
Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Aim: Knowledge-focused
Last updated Jul 02, 2026 00:00 UTC