Avidity Biosciences Says Data Suggest Del-zota Has Potential To Change Treatment Paradigm And Course Of Disease For Patients With Duchenne Muscular Dystrophy Mutations Amenable To Exon 44 Skipping
Avidity Biosciences Says Data Suggest Del-zota Has Potential To Change Treatment Paradigm And Course Of Disease For Patients With Duchenne Muscular Dystrophy Mutations Amenable To Exon 44 Skipping
AOC 1044 demonstrated favorable safety and tolerability. Avidity also announced delpacibart zotadirsen as the approved international nonproprietary name of AOC 1044, abbreviated as del-zota.
AOC 1044表现出良好的安全性和耐受性。Avidity还宣布将AOC 1044的批准国际非专有名称命名为delpacibart zotadirsen，缩写为del-zota。
Statistically significant increase of 25% of normal in dystrophin production and restored total dystrophin up to 54% of normal with delpacibart zotadirsen 5 mg/kg at four months
使用delpacibart zotadirsen 5 mg/kg治疗四个月， dystrophin产量正常值增加了25%，恢复了总的dystrophin达到正常值的54%。
Diana Castro, M.D., Board Certified Neurologist and Neuromuscular Physician, Founder and Director Neurology and Neuromuscular Care Center, Founder and Director Neurology Rare Disease Center and EXPLORE44 trial program investigator, said, "These del-zota data are very encouraging as children and adults living with Duchenne muscular dystrophy mutations amenable to exon 44 skipping are still in need of targeted treatment options to address this debilitating disease."
Diana Castro万. D.，董事会认证神经学家和神经肌肉科医生，神经学和神经肌肉护理中心创始人和主任，神经学罕见病中心创始人和EXPLORE44试验项目调查员说："这些del-zota数据非常令人鼓舞，因为患有Duchenne肌萎缩症突变的儿童和成年人仍需要有针对性的治疗选择来治疗这种令人沮丧的疾病。"