https://www.miplyffa-hcp.com/ 2025-05-09 https://www.miplyffa-hcp.com/wp-content/themes/NCS_template/images/logo/MIPLYFFA.svg MIPLYFFA HCP website homepage. https://www.miplyffa-hcp.com/wp-content/themes/NCS_template/images/logo/zevra-white.svg Zevra website. https://www.miplyffa-hcp.com/about-npc 2025-05-09 https://www.miplyffa-hcp.com/wp-content/themes/NCS_template/images/NPC-Age-Presentation.svg Chart showing how symptoms, which are classified as visceral-neurodegenerative, neurodegenerative, and psychiatric-neurodegenerative, progress and present differently depending on age. https://www.miplyffa-hcp.com/npc-diagnosis 2025-05-09 https://www.miplyffa-hcp.com/wp-content/themes/NCS_template/images/ambulation.svg Ambulation domain symptoms range from clumsiness to wheelchair dependence. https://www.miplyffa-hcp.com/wp-content/themes/NCS_template/images/fine.svg Fine motor skills domain symptoms range from slight dysfunction to severe muscle impairment. https://www.miplyffa-hcp.com/wp-content/themes/NCS_template/images/swallow.svg Swallow domain symptoms range from a cough while eating to feeding tube dependence. https://www.miplyffa-hcp.com/wp-content/themes/NCS_template/images/speech.svg Speech domain symptoms range from slurred or slow speech to no communication. https://www.miplyffa-hcp.com/clinical-trials 2025-05-09 https://www.miplyffa-hcp.com/wp-content/themes/NCS_template/images/observational.svg Primary endpoint: Change from baseline in the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS). https://www.miplyffa-hcp.com/wp-content/themes/NCS_template/images/NPC-symptom-reduction-chart.svg Chart showing NPC symptom reduction at 12 months with MIPLYFFA with miglustat in comparison to placebo with miglustat, as measured by the R4DNPCCSS score. https://www.miplyffa-hcp.com/safety 2025-05-09 https://www.miplyffa-hcp.com/wp-content/themes/NCS_template/images/AdverseReactions.svg Chart showing adverse reactions with MIPLYFFA and miglustat in comparison to placebo and miglustat. The most common adverse reactions were upper respiratory tract infection and diarrhea. https://www.miplyffa-hcp.com/dosing 2025-05-09 https://www.miplyffa-hcp.com/wp-content/themes/NCS_template/images/RecommendedDoseChart.svg Recommended MIPLYFFA dose (3 times per day) for patients aged 2 years and older by body weight: 47 mg for 18-33 lb (8-15 kg), 62 mg for >33-60 lb (>15-30 kg), 93 mg for >60-121 lb (>30-55 kg), and 124 mg for >121 lb (>55 kg). https://www.miplyffa-hcp.com/resources 2025-05-09 https://www.miplyffa-hcp.com/wp-content/themes/NCS_template/images/amplifyassist.svg Zevra AmplifyAssist website. https://www.miplyffa-hcp.com/contact-a-rep 2025-05-09 https://www.miplyffa-hcp.com/documents/miplyffa-hcp-fact-sheet.pdf 2025-05-09