Healthcare professionals may assess symptoms by focusing on “domains” or areas impacted by NPC within a validated scale or measuring system. The scale includes scores for how serious the symptoms are for an individual, and can measure how they have changed over time.
The R4DNPCCSS is a validated scale for NPC that measures the 4 domains, shown below, that have been identified as some of the most important by NPC expert clinicians, caregivers, and patients, and allows for an assessment of NPC symptom progression.
These scales also provide a way to demonstrate treatment efficacy and see how you or your loved one may be responding to treatment. Download the NPC Assessment Guide for more details.
MIPLYFFA is prescription medicine used in combination with a drug called miglustat to treat neurological symptoms of Niemann-Pick disease type C (NPC) in patients 2 years of age and older.
Before starting MIPLYFFA, tell your healthcare provider about all your medical conditions, including if you are pregnant or plan to become pregnant, breastfeeding or plan to breastfeed.
Tell your healthcare provider about all the medicines you take, including any prescription and over-the-counter medicines, vitamins, or herbal supplements. MIPLYFFA may affect how other medicines work.
What are the possible side effects of MIPLYFFA?
MIPLYFFA may cause serious side effects including:
The most common side effects of MIPLYFFA in patients also taking miglustat include upper respiratory tract infection, diarrhea and decreased weight.
These are not all the possible side effects of MIPLYFFA. Call your HCP for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch., or call 1-800-FDA-1088.
Drug Interactions: MIPLYFFA can cause side effects if used together with certain drugs called OCT2 substrates. Talk to your healthcare provider about any drugs that you may be taking for other conditions.
MIPLYFFA capsules for oral use are available in the following strengths in a 90-count bottle: 47 mg, 62 mg, 93 mg, and 124 mg.
For more information, please see the Prescribing Information, including Instructions for Use.