HealthQuill Autosomal Recessive Pattern
The US Food and Drug Administration cleared PTC Therapeutics’ sepiapterin for treating children and adults with a rare genetic condition where the body cannot break down a part of protein called phenylalanine, which comes from foods like meat, eggs, and milk.
Drugs Health Pharma

US regulator clears PTC’s medicine to treat a rare genetic metabolic disease 

HQ Team July 29, 2025: The US Food and Drug Administration cleared PTC Therapeutics’ sepiapterin for treating children and adults with a rare.

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