Shares of Rocket Pharmaceuticals, Inc. (RCKT) surged over 40% on Wednesday morning after the company announced that alignment has been reached with the FDA on the global Phase 2 pivotal trial of RP-A501 for Danon Disease.RCKT is currently trading at $21.61, up $6.32 or 41.33%, on a huge volume of 15 million shares, above average volume of 600 thousand, on the Nasdaq.

在该公司宣布与FDA就Danon Disease的RP-A501全球第二阶段关键试验达成一致后，Rocket Pharmaceuticals，Inc。（RCKT）的股份在周三早上飙升超过40%.RCKT目前的交易额为21.61美元，在纳斯达克股票代码上大幅增加1500万股，超过平均60万股，上涨6.32美元或41.33%。

The stock opened its trading at $21.44 after closing Tuesday's trading at $15.29. The stock has traded between $11.78 and $24.53 in the past 52-week period.RP-A501 is Rocket's investigational gene therapy product for the treatment of Danon Disease and the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies..

该股票在星期二以15.29美元的交易结束后以21.44美元开盘。该股票在过去52周内交易量在11.78美元至24.53美元之间。RP-A501是火箭公司用于治疗达农病的研究性基因治疗产品，也是第一个在临床研究中证明安全性和有效性的心血管疾病基因治疗产品。。

Danon Disease is a uniformly fatal inherited cardiomyopathy that leads to mortality in the majority of male patients at age ~20 and females at age ~40, and for which there are no approved curative or disease-modifying therapies. The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe.The global, single-arm, multi-center Phase 2 pivotal trial will evaluate the efficacy and safety of RP-A501 in 12 patients with Danon Disease, including a pediatric safety run-in (n=2), with a natural history comparator and a dose level of 6.7 x 1013 GC/kg.

达农病是一种致命的遗传性心肌病，可导致20岁左右的大多数男性患者和40岁左右的女性死亡，并且尚无批准的治疗方法或可改善疾病的疗法。该疾病影响美国和欧洲约15000至30000名患者。全球单臂多中心2期关键试验将评估RP-A501对12名Danon病患者的疗效和安全性，包括儿科安全性磨合（n=2），具有自然史比较和6.7 x 1013 GC/kg的剂量水平。

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