METHYLMALONIC ACIDEMIA (MMA)
Clinical trials for METHYLMALONIC ACIDEMIA (MMA) explained in plain language.
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New hope for kids with rare metabolic disease
Disease control Not yet recruitingThis study is testing an injection called hydroxocobalamin to see if it can safely control a rare, inherited disorder in children. The disorder causes harmful chemicals to build up in the body. Researchers will measure if the treatment can bring these chemical levels back to norm…
Matched conditions: METHYLMALONIC ACIDEMIA (MMA)
Phase: PHASE3 • Sponsor: CSPC ZhongQi Pharmaceutical Technology Co., Ltd. • Aim: Disease control
Last updated Mar 16, 2026 15:25 UTC
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Scientists track rare childhood disease to pave way for future cures
Knowledge-focused Not yet recruitingThis 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…
Matched conditions: METHYLMALONIC ACIDEMIA (MMA)
Sponsor: Genespire Srl • Aim: Knowledge-focused
Last updated Mar 23, 2026 15:17 UTC