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treatment of muskeldystrophien and related disorders

机译:肌肉营养不良和相关疾病的治疗

摘要

The invention provides, among other aspects, compositions and methods for treating, preventing, and diagnosing diseases or conditions associated with an abnormal level or activity of biglycan; diseases or conditions associated with an abnormal level or activity of collagen VI; disorders associated with an unstable cytoplasmic membrane, due, e.g., to an unstable dystrophin associated protein complex (DAPC); and disorders associated with abnormal synapses or neuromuscular junctions, including those resulting from an abnormal MuSK activation or acetylcholine receptor (AChR) aggregation.
机译:除了其他方面,本发明提供了用于治疗,预防和诊断与双链聚糖的异常水平或活性有关的疾病或病症的组合物和方法;与胶原VI水平或活性异常有关的疾病或状况;与不稳定的细胞质膜有关的疾病,例如由于不稳定的肌营养不良蛋白相关蛋白复合物(DAPC);与异常突触或神经肌肉接头有关的疾病,包括由异常的MuSK激活或乙酰胆碱受体(AChR)聚集引起的疾病。

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