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Long-term carglumic acid therapy in adult siblings with methylmalonic acidemia and advanced chronic kidney disease

Article C Peer reviewed Observational

Key takeaways

This report describes two adult siblings with a rare metabolic condition and advanced kidney disease. They took a drug called carglumic acid for a long time. The study tracks their clinical course and how the drug helped manage their condition. It is a case report, so it shows what happened in these specific people, not a general proof.

Who did this work

Yoo-Mi Kim · Han Hyuk Lim · Eunhee Kim · Geena Kim · Jae Wan Jeon · Hae Ri Kim · Jong Seok Joo

Source

Article · OpenAlex, Kidney disease concept sweep (verified C2778653478) · 2026-10-07
https://doi.org/10.1186/s13023-026-04637-3