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Marc Engelen1, Stephan Kemp2, Florian Eichler3
1Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam UMC, Vrije Universiteit, Amsterdam, The Netherlands; Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Vrije Universiteit, Amsterdam, The Netherlands.
链接X的 adrenoleukodystrophy (ALD) 是一种影响脂肪酸代谢的遗传疾病. 虽然造血细胞移植可以阻止大脑ALD,但肌肉病的治疗方法仍然有限.
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