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While the condition is readily visible at birth, many times the deformity is detected on prenatal ultrasounds, allowing weeks or months for the parents to prepare for the expected treatment

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By targeting phenotype and complication clusters, ranging from cardiovascular to hepatic and behavioral axes, clinicians can tailor pharmacologic regimens to achieve disease modification, symptom relief, and prevention

It needs a massive supply of antioxidants to protect its own tissues while it breaks down harmful substances

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