PTC Therapeutics, Inc. (NASDAQ: PTCT) announced the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene therapy approved in the United States that is directly administered to the brain. The gene therapy, which will be marketed in the United States with the brand name KEBILIDI™ (eladocagene exuparvovec-tneq), is indicated for the treatment of children and adults with AADC deficiency, including the full spectrum of disease severity. Launch preparations are well underway, with centers of excellence already identified and surgeons trained in the procedure to deliver the gene therapy. AADC deficiency is a highly morbid and life-shortening rare genetic disorder that results in the inability to synthesize dopamine, a neurotransmitter essential for motor function. KEBILIDI is a gene replacement therapy that is directly administered to the putamen of the… 

Implantica AG (publ.) 是一家处于将先进技术引入人体的最前沿的医疗技术公司，其独特设备RefluxStop™用于治疗酸反流，这是一个涉及10亿患者的治疗领域，该公司宣布已向美国FDA提交RefluxStop™的上市前批准（PMA）申请的第二个临床模块，并附带对FDA从第一个模块中发现的问题的答复。本周早些时候提交的用于RefluxStop™ PMA的第二个模块是FDA批准的三模块流程中最关键的模块。我们提交给FDA的临床研究结果显示出极好的积极结果，这在外科界引起了广泛反响，激发了全球（尤其是在美国）的外科医生和胃肠病专家对RefluxStop™的强烈兴趣。与模块2一起提交的还有对FDA从模块1中发现的问题的答复…… 

Suvoda是一家全球临床试验技术公司，专注于肿瘤学、中枢神经系统（CNS）和罕见病等治疗领域的复杂研究。该公司宣布已收到美国专利局颁发的专利，授予其eCOA（电子临床结果评估）产品的软件架构的一部分——Suvoda问卷定义语言（SQDL）。该专利证明了Suvoda eCOA的强大功能：加速问卷的创建、翻译、本地化和部署，使临床试验申办方能够更高效地启动其研究。虽然eCOA传统上是临床试验实施中的一个瓶颈，但Suvoda eCOA及其SQDL工具使申办方能够在数小时内而不是数天内提供高质量的问卷。