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Site directed and saturation mutagenesis were performed using the Quikchange II kit (Agilent Technologies) or by overlapping PCR

This dose is maintained for at least 4 weeks, depending on patient tolerance and treatment goals

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In typical neonatal cases of PA, symptoms usually appear within hours to days after birth and are often accompanied by acute metabolic disturbances, poor general condition, feeding difficulties, vomiting, dehydration and temperature instability