Mucopolysaccharidosis Type I in Mexico: Case-Based Review.
Children (Basel, Switzerland)(2023)
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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