Scales
- mFARS, SARA, ICARS
Therapy
- Omaveloxolone (Skyclarys)
Approval 2023 (MOXIE 1&2 trials) in FA > 16yo
> MOA: red NRF2 transcription
> start 50 mg (after 2wks) ➡️ 100 (+2 wks) ➡️ 150
> monitor LFTs (start & qmo), BNP, and lipids
If inc BNP or CHF symptoms, stop med and investigate ADHF, order TTE
> no per feeding tube, but can give open sprinkle pill in apple sauce
> avoid grapefruit
> no pregnancy studies