https://www.miplyffa.com/ 2025-05-09 https://www.miplyffa.com/wp-content/themes/NCS_template/images/logo/MIPLYFFA.svg MIPLYFFA Homepage. https://www.miplyffa.com/wp-content/themes/NCS_template/images/logo/zevra-white.svg ZEVRA.com website. https://www.miplyffa.com/what-is-npc/ 2025-05-09 https://www.miplyffa.com/wp-content/themes/NCS_template/images/Lysosome.svg Gene mutations lead to a buildup of lipids (fats) in the lysosomal cells of the body. https://www.miplyffa.com/wp-content/themes/NCS_template/images/ambulation.svg Ambulation symptoms range from clumsiness to wheelchair dependence. https://www.miplyffa.com/wp-content/themes/NCS_template/images/fine.svg Fine motor skills symptoms range from slight dysfunction to severe muscle impairment. https://www.miplyffa.com/wp-content/themes/NCS_template/images/swallow.svg Swallowing difficulties range from coughing while eating to feeding tube dependence. https://www.miplyffa.com/wp-content/themes/NCS_template/images/speech.svg Speech symptoms range from slurred or slow speech to no communication. https://www.miplyffa.com/about-miplyffa/ 2025-05-09 https://www.miplyffa.com/wp-content/themes/NCS_template/images/HowToTake.svg One capsule 3 times per day, with or without food. https://www.miplyffa.com/clinical-trials/ 2025-05-09 https://www.miplyffa.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.com/taking-miplyffa/ 2025-05-09 https://www.miplyffa.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.com/financial-support/ 2025-05-09 https://www.miplyffa.com/wp-content/themes/NCS_template/images/amplifyassist.svg Zevra AmplifyAssist website. https://www.miplyffa.com/documents/miplyffa-caregiver-fact-sheet.pdf 2025-05-09