Highly effective modulator therapy (HEMT) for cystic fibrosis (CF) has been touted as one of the greatest advances to date in CF care. As these therapies are now available for many older children and adults with CF, marked improvement of their nutritional status, pulmonary and gastrointestinal symptoms has been observed. However, most infants and younger children are not current candidates for HEMT due to age and/or cystic fibrosis transmembrane conductance regulator (CFTR) mutation. For these young children, it is essential to provide rigorous monitoring and care to avoid potential disease sequelae while awaiting HEMT availability. The following article highlights recent advances in the care of infants and young children with CF with regard to surveillance and treatment of nutritional, pulmonary, and gastrointestinal disorders. Recent clinical trials in this population are also reviewed.

中文翻译：

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囊性纤维化早期治疗的最新进展：缩小与高效调节剂治疗的差距

囊性纤维化 (CF) 的高效调节剂疗法 (HEMT) 被吹捧为迄今为止 CF 护理中最大的进步之一。由于这些疗法现在可用于许多患有 CF 的年龄较大的儿童和成人，因此观察到他们的营养状况、肺部和胃肠道症状有显着改善。然而，由于年龄和/或囊性纤维化跨膜电导调节器 (CFTR) 突变，大多数婴儿和年幼儿童目前不适合 HEMT。对于这些年幼的儿童，在等待 HEMT 可用时，必须提供严格的监测和护理以避免潜在的疾病后遗症。以下文章重点介绍了 CF 婴幼儿护理在营养、肺部和胃肠道疾病的监测和治疗方面的最新进展。