New drug for Niemann Pick disease type C appears safe effective

New drug for Niemann-Pick disease type C1 appears safe, effective

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Aug. 11 (UPI) — Researchers report that an experimental drug appears to be safe and effective at slowing the progression of Niemann-Pick disease type C1.

Niemann-Pick disease type C1 is a rare, fatal genetic disorder that affects children and teens causing progressive decline in cognitive and neurological function.

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