Scientists track rare childhood disease to pave way for future cures
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This study aims to better understand how a rare and severe genetic disorder called Methylmalonic Acidemia (MMA) progresses in children. Researchers will follow up to 30 children, aged 16 or younger, who have a severe form of the disease, with or without a liver transplant. The go…
Sponsor: Genespire Srl • Aim: Knowledge-focused
Last updated Mar 23, 2026 15:17 UTC