FDA approves first drug for Thymidine Kinase 2 Deficiency: All we know about this very rare mitochondrial disease

A groundbreaking FDA approval offers the first-ever treatment for ultra-rare Thymidine Kinase 2 Deficiency (TK2d). KYGEVVI, a combination therapy, provides hope for children and adults by addressing impaired mitochondrial DNA production. This milestone marks a significant advancement in mitochondrial medicine, potentially transforming care for those with this devastating genetic disorder.

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