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FDA grants Rare Pediatric Disease designation to Thiogenesis' drug for Leigh syndrome

Market News
13 Jul 2026
Newsfile Corp
View Source
Bullish
pluang ai news

Thiogenesis Therapeutics received Rare Pediatric Disease designation from the FDA for its drug candidate TTI-0102, targeting Leigh syndrome, a serious mitochondrial disorder affecting children. This designation may allow the company to obtain a Priority Review Voucher upon drug approval, which can expedite future drug reviews or be sold. Thiogenesis plans to start a Phase 2 clinical trial soon, incorporating insights from previous studies to improve the treatment's effectiveness and safety. This milestone highlights the company's progress in addressing unmet medical needs in rare pediatric diseases.

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