Zevra Therapeutics Media Release | April 15, 2024
Zevra Therapeutics Presents New Data On The Long-Term Safety And Efficacy Of Arimoclomol As A Treatment For Niemann-Pick Disease Type C At The SIMD 45th Annual Meeting
Read the full statement from Zevra Therapeutics here:
Here is a summary of the article:
Clinical Benefits: The data from the Expanded Access Program showed a consistent safety profile and a clinically meaningful slowing of disease progression in adults with NPC.
Regulatory Progress: The FDA has accepted the resubmission of the NDA for Arimoclomol with a PDUFA date set for September 21, 20242.
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