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中文摘要
翻译
脊髓损伤(SCIs)会导致身体的部分或大部分瘫痪和感觉丧失。许多sci患者是永久性残疾,一生都依赖轮椅和其他辅助设备。脊髓损伤的完全恢复通常是不可能的,因为脊髓的轴突通常是受损的,在成人中不能再生。
英文摘要
Spinal cord injuries (SCIs) cause paralysis and loss of feeling in some or most of the body. Many people with SCIs are permanently disabled, relying on wheelchairs and other assistive devices for their entire lives. Complete recovery from SCI is normally not possible because the axons of the spinal cord are usually damaged and cannot regrow in adults. The lead collaborators have developed a therapeutic candidate (AXER-204) that stimulates corticospinal and raphespinal axon growth. Additionally, it has been shown that functional recovery can be achieved in acute, subacute and chronic spinal contusion. Preclinical studies that demonstrate efficacy in an animal model of chronic SCI provide the basis for launching clinical trials of AXER-204. This projects aim is to further develop this therapy to prepare it for testing in human clinical trials. The BrIDGs team is collaborating on the completion of the following studies on AXER-204: - Synthesis of non-GMP and Good Manufacturing Practice (GMP) material - Formulation development - Pharmacokinetic/absorption, distribution, metabolism, and excretion (PK/ADME) studies - IND-directed toxicology
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