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Rather than simply discontinuing medication and hoping for the best, participants underwent a procedure called duodenal mucosal resurfacing (DMR), performed using an investigational device called Revita

The FDA issued warning letters in March 2026 (FDA.gov press announcement, March 3, 2026) specifically targeting companies that implied otherwise

Limited data has been reported on the burden and impact of approved on-demand therapies on patient and caregiver health-related quality of life (HRQoL)

Carnitine membrane transporter deficiency: a long-term follow up and OCTN2 mutation in the first documented case of primary carnitine deficiency