The global Leigh syndrome treatment market is witnessing high growth owing to the advancement in gene therapy. Leigh syndrome is a rare neurological genetic disorder that affects the central nervous system and metabolism. It is usually seen in infants and children under the age of 2. The signs and symptoms associated with Leigh syndrome include poor muscle tone, muscle weakness, vision issues, lack of coordination, impaired gait, breathing difficulties, and organ dysfunction. The treatment options are mainly focused on symptom management through physical therapy, medications, surgical intervention, and oxygen therapy. However, gene therapy offers promising results for treating the underlying cause of the disease.

The Global Leigh syndrome treatment market is estimated to be valued at US$ 272.9 MN in 2024 and is expected to exhibit a CAGR of 6.7% over the forecast period 2024 to 2031.

Key Takeaways
Key players operating in the Global Leigh Syndrome Treatment Market Growth are Abliva AB, PTC Therapeutics, VAKS Pharma, MITOCH, Medley Pharmaceuticals Ltd., Khondrion BV, OMEICOS THERAPEUTICS GMBH. , Edison Pharmaceuticals, Inc. , Dainippon Sumitomo Pharma Co. Ltd., Taysha GTx, PicnicHealth (AllStripes), Takeda Pharmaceutical Company , Biogen, Ionis Pharmaceuticals, Inc., Sarepta Therapeutics, Inc., and MECOSON LABS PRIVATE LIMITED.

The key opportunities in the market include a large patient pool suffering from Leigh syndrome, increasing research funding for developing novel therapies, and growing awareness about gene therapy. Advancement in gene therapy using genetic constructs, vectors, and delivery techniques offer promising results to treat the root cause of the disease.

Market Drivers
The major market drivers include the rising prevalence of rare genetic diseases like Leigh syndrome, growing demand for effective treatments, availability of government support and funding for research, and increasing clinical trial activities. According to the estimates of the United Mitochondrial Disease Foundation, the prevalence of Leigh syndrome in the United States ranges between 1 in 30,000 to 1 in 40,000 births.

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