9月3日 - ** Ultragenyx Pharmaceutical（RARE.O）股价在盘前交易中下跌45.5%，至14.4美元
** 该公司研发的药物阿帕祖纳森（apazunersen） (link) 在针对安杰尔曼综合征（一种影响神经系统并导致儿童期大脑发育异常的疾病）的晚期临床试验中未能达到主要研究目标
** 与安慰剂相比，该药物在改善儿童认知能力或整体发育方面未显示出显著效果
** Ultragenyx表示正在评估apazunersen项目的未来走向
** 此次试验结果“毫无疑问是负面的”，此次失败“增加了竞争对手的风险”，例如Ionis（IONS.O），该公司目前正在进行自己的类似安杰尔曼综合征晚期临床试验
** 截至上一交易日收盘，该股年内累计上涨15.4%
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