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表現出良好的安全性和耐受性。Avidity還宣佈將AOC 1044的批准國際非專有名稱命名爲delpacibart zotadirsen，縮寫爲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治療四個月， dystrophin產量正常值增加了25%，恢復了總的dystrophin達到正常值的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萬. D.，董事會認證神經學家和神經肌肉科醫生，神經學和神經肌肉護理中心創始人和主任，神經學罕見病中心創始人和EXPLORE44試驗項目調查員說："這些del-zota數據非常令人鼓舞，因爲患有Duchenne肌萎縮症突變的兒童和成年人仍需要有針對性的治療選擇來治療這種令人沮喪的疾病。"