在Charcot-Marie-Tooth疾病的临床试验:回顾性和临床前评估

Malavika A Nair1, Zhiyv Niu2,3, Nicholas N Madigan4

  • 1Department of Graduate Education, Alix School of Medicine, Rochester, MN, United States.

Frontiers in neurology
|October 9, 2023
PubMed
概括

针对Charcot-Marie-Tooth (CMT) 疾病的临床试验显著增加,重点是新型基因疗法和程序治疗. 虽然许多有前途的分子疗法仍处于临床前阶段,但人类试验正在推进,显示整个行业的参与.

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