INDIANAPOLIS (WISH) — Niemann-Pick Type C, or NPC, is a relentlessly progressive and fatal genetic disorder that impacts both the body and the brain. In the U.S., only about 900 people are living with ...
A Zevra Therapeutics drug is now the first FDA-approved therapy for an ultra-rare, inherited metabolic disorder whose effects on the central nervous system can become fatal by the time a patient is a ...
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