Zevra Therapeutics is hoping to hit the ground running with its Niemann-Pick disease type C (NPC) therapy arimoclomol in the autumn if it gets through an advisory committee with US regulators.
BIO: Upbeat Zevra Advances Promising Rare Disease Portfolio
With the urea cycle disorder treatment Olpruva launched and the NPC therapy arimoclomol closing in on US approval, Zevra is one to watch in the rare disease space.
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