FDA 批准基因疗法 Lenmeldy 用于治疗儿童早发性异染性脑白质营养不良。 FDA approves gene therapy Lenmeldy for early-onset metachromatic leukodystrophy in children.
FDA 已批准 Lenmeldy（atidarsagene autotemcel），这是第一个用于治疗患有早发性异染性脑白质营养不良（MLD）儿童的基因疗法，MLD 是一种影响大脑和神经系统的罕见遗传病。 The FDA has approved Lenmeldy (atidarsagene autotemcel), the first gene therapy for the treatment of children with early-onset metachromatic leukodystrophy (MLD), a rare genetic disease affecting the brain and nervous system. 该批准涵盖了疾病出现症状前的婴儿晚期、症状出现前的青少年早期和症状出现前的青少年早期阶段的儿童。 The approval covers children in the pre-symptomatic late infantile, pre-symptomatic early juvenile, and early symptomatic early juvenile stages of the disease. Lenmeldy 是一种一次性、个体化的单剂量输注剂，由患者自身的造血干细胞制成，这些干细胞经过基因改造，包含 ARSA 基因的功能副本。 Lenmeldy is a one-time, individualized single-dose infusion made from a patient's own hematopoietic stem cells, which have been genetically modified to include functional copies of the ARSA gene.