Zevra Therapeutics announced the publication of positive phase 2/3 data on MIPLYFFA (arimoclomol) in infants aged 12 to under 24 months with Niemann-Pick disease type C (NPC). The study showed that MIPLYFFA was well tolerated with no new safety concerns and pharmacokinetics consistent with older children. This supports the potential for earlier treatment initiation in this rare, progressive neurodegenerative disease. Zevra continues to expand clinical evidence and regulatory efforts for MIPLYFFA, which is already FDA-approved for NPC in patients aged 2 and older.
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Iran has warned it will retaliate fiercely if the US targets its infrastructure, following President Trump's threat to strike Iranian power plants and bridges next week if negotiations fail. The US recently conducted airstrikes against Iranian milita...
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