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NIH announces start of clinical trial using cyclodextrin as a potential treatment for Niemann-Pick disease type C1 — a rare, fatal neurological disorder. The collaborative Phase I trial, which stems from pilot work pursued by the TRND program, will test the drug in nine patients.

Pre-clinical scientists at NCATS have also published promising work on another treatment candidate for Niemann-Pick type C1: delta-tocopherol, a form of vitamin E.


Learn how you can participate in clinical trials - National Institutes of Health