Mucopolysaccharidosis Type I in Mexico: Case-Based Review.

Children (Basel, Switzerland)(2023)

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摘要
Despite the challenges of managing this rare disease in Mexico, our patient benefited from the combined therapy. The discrete clinical manifestations and prompt evaluation by a geneticist were crucial in establishing a diagnosis, enabling an early intervention by a multidisciplinary team. The combination of ERT before and after HSCT provided health benefits to our patient.
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case-based
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