Zevra Therapeutics Announces Publication of Positive MIPLYFFA® Data from Pediatric Substudy in Infants with Niemann-Pick Disease Type C (NPC)
Zevra Therapeutics announced the publication of positive data from a Phase 2/3 open-label pediatric substudy evaluating MIPLYFFA in infants with Niemann-Pick disease type C. The study, which included five children aged 12 to <24 months, found that arimoclomol was generally well tolerated with no new safety signals and pharmacokinetic results consistent with older pediatric populations.
Key figures
- Drug Name
- MIPLYFFA (arimoclomol)
- Phase Of Trial
- Phase 2/3
- Sample size
- 5
- Age range months
- 12 to <24
- Treatment duration months
- up to 36
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