Methylmalonic Acidemia (MMA): Therapeutic Progress, Market Evolution, and Future Prospects
Overview of Methylmalonic Acidemia and Its Clinical Burden Methylmalonic Acidemia (MMA) is a rare genetic metabolic disorder resulting from the malfunction of the enzyme methylmalonyl-CoA mutase, which is crucial for breaking down specific amino acids and fatty acids. This enzymatic defect leads to the accumulation of toxic metabolites, causing multi-organ complications, particularly affecting...
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