Thymidine Kinase 2 Deficiency (TK2D) is a progressive mitochondrial disorder characterized by severe muscle weakness and respiratory impairment. The therapeutic landscape is evolving rapidly, fueled by increasing R&D investment in rare diseases and breakthroughs in genetic medicine. Industry leaders such as Modis Therapeutics, Ultragenyx, and ReGenX Biosciences are pioneering novel treatment modalities to address this critical unmet medical need.

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