As of January 2026, the China Leigh Syndrome treatment market is identifying as a specialized but rapidly accelerating segment of the rare disease sector, with a valuation projected to reach approximately $10 million this year. The 2026 landscape is defined by the peak implementation of the National Rare Disease Registry System, which has identified over 1,500 Leigh Syndrome cases across China,...

Leigh Syndrome, also known as Leigh disease, is a rare, progressive, and debilitating neurodegenerative disorder primarily affecting infants and young children. Characterized by defects in mitochondrial energy production, the syndrome leads to severe neurological dysfunction, muscle weakness, developmental delays, and respiratory complications. Due to its genetic and metabolic complexity,...

4 gene therapy trials showing curative potential for hepatic copper in 2026 As 2026 begins, the global hepatology community is closely monitoring a wave of breakthrough data from Phase II and III trials that aim to permanently correct the ATP7B genetic mutation. Regulatory authorities in the European Union and the United States have recently signaled a willingness to accept biomarker-driven...