ClearPoint Neuro的装置 得到FDA的批准 提供基因治疗 以治疗罕见的脑部疾病 AADC缺乏症 ClearPoint Neuro's device gets FDA approval to deliver gene therapy for a rare brain disorder, AADC deficiency.
ClearPoint Neuro已获得林业发展局批准其SmartFlow Neuro Cannula(SmartFlow Neuro Cannula),这是第一个被批准为AADC缺乏症(一种罕见的遗传障碍)直接向大脑提供基因疗法的装置。 ClearPoint Neuro has received FDA approval for its SmartFlow Neuro Cannula, the first device approved to deliver gene therapy directly to the brain for AADC deficiency, a rare genetic disorder. 该设备用于管理PTC治疗学科的KEBILIDI基因疗法,其目的是恢复多巴胺的生产,改善病人的运动功能。 The device is used to administer PTC Therapeutics' KEBILIDI gene therapy, which aims to restore dopamine production, improving motor function in patients. 这标志着神经基因疗法的重大进步,为美国AADC缺乏症提供了第一种疾病调控疗法。 This marks a significant advancement in neuro gene therapy, offering the first disease-modifying treatment for AADC deficiency in the U.S.