Zevra Therapeutics Launches MIPLYFFA for Niemann-Pick Disease

An announcement from Zevra Therapeutics ( (ZVRA) ) is now available.

Zevra Therapeutics has announced the commercial availability of MIPLYFFA™ (arimoclomol), the first FDA-approved treatment for Niemann-Pick Disease Type C (NPC). This breakthrough offers a new hope for patients, as it is used in combination with miglustat to treat neurological symptoms in both adults and children. Zevra’s patient support program, AmplifyAssist™, provides essential resources to navigate prescription processes and insurance challenges, ensuring patients can access this life-changing therapy.

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