Methylmalonic Acidemia (MMA): Shifting Therapeutic Landscape and Market Evolution
Overview of Methylmalonic Acidemia (MMA) and Its Clinical Burden Methylmalonic Acidemia (MMA) is a rare genetic metabolic disorder caused by impaired activity of the enzyme methylmalonyl-CoA mutase, which is necessary for breaking down specific proteins and fats. This dysfunction results in the accumulation of toxic metabolites, leading to multi-organ complications affecting the brain, kidneys,...
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