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For example, an orphan drug designation for AAV9hPCCA for the treatment of propionic acidemia was granted by the FDA to the National Center for Advancing Translational Sciences in May 2022
N Engl J Med 361 , 115263 (2009)
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As such, addressing these areas is a good place to start
Thus, it acts as a buffer by inhibiting the accumulation of lactic acid in the muscles, which helps delay fatigue and prevent cramping
