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治疗肌肉营养不良症有效的首个实验性药物
作者:网络来源 时间:2012-10-15 点击:391 来源:网络来源
<P style="MARGIN: 0cm 0cm 0pt; TEXT-INDENT: 24pt; TEXT-ALIGN: left; mso-pagination: widow-orphan; mso-line-height-alt: 0pt; mso-char-indent-count: 2.0" align=left><SPAN style="FONT-SIZE: 12pt; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-font-kerning: 0pt; mso-ansi-language: en; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial"><SPAN style="FONT-SIZE: 12pt; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-font-kerning: 0pt; mso-ansi-language: en; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial"></SPAN>据福布斯杂志</SPAN> <SPAN lang=EN style="FONT-SIZE: 12pt; FONT-FAMILY: Arial; mso-font-kerning: 0pt; mso-ansi-language: en" XML:LANG="EN">10</SPAN><SPAN style="FONT-SIZE: 12pt; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-font-kerning: 0pt; mso-ansi-language: en; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">月</SPAN><SPAN lang=EN style="FONT-SIZE: 12pt; FONT-FAMILY: Arial; mso-font-kerning: 0pt; mso-ansi-language: en" XML:LANG="EN">3</SPAN><SPAN style="FONT-SIZE: 12pt; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-font-kerning: 0pt; mso-ansi-language: en; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">日</SPAN><SPAN style="FONT-SIZE: 12pt; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-font-kerning: 0pt; mso-ansi-language: en; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">报道:一种</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">新的实验性药物看来能改善肌肉营养不良(或肌肉萎缩)症男孩的肌肉功能。在用药</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">1</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">年后,与以前相比他们在</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">6</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">分钟内行走的距离增加。</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US"><BR><SPAN style="mso-spacerun: yes"><FONT face=Arial><FONT color=#333333>&nbsp;<WBR>&nbsp;<WBR>&nbsp;<WBR></FONT></FONT></SPAN></SPAN> <SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">这些结果是</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">“</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">令人难以置信<SPAN><FONT color=#333333>,</FONT></SPAN></SPAN><SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">”</SPAN></SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">美国宾夕法尼亚大学</SPAN><SPAN lang=EN style="FONT-SIZE: 12pt; FONT-FAMILY: Arial; mso-font-kerning: 0pt; mso-ansi-language: en" XML:LANG="EN">Perelman</SPAN><SPAN style="FONT-SIZE: 12pt; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-font-kerning: 0pt; mso-ansi-language: en; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">医学院生理学负责人</SPAN><SPAN lang=EN style="FONT-SIZE: 12pt; FONT-FAMILY: Arial; mso-font-kerning: 0pt; mso-ansi-language: en" XML:LANG="EN">Lee Sweeney</SPAN><SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">说。他专门从事</SPAN></SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">肌肉萎缩症研究,没有参与这项科研工作。</SPAN></P> <P style="MARGIN: 0cm 0cm 0pt; TEXT-INDENT: 24pt; TEXT-ALIGN: left; mso-pagination: widow-orphan; mso-line-height-alt: 0pt; mso-char-indent-count: 2.0" align=left><SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">“患者</SPAN></SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">一旦发病就会每况愈下,根本不会改善。”</SPAN></P> <P style="MARGIN: 0cm 0cm 0pt; TEXT-INDENT: 24pt; TEXT-ALIGN: left; mso-pagination: widow-orphan; mso-line-height-alt: 0pt; mso-char-indent-count: 2.0" align=left><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">杜氏(</SPAN><SPAN lang=EN style="FONT-SIZE: 12pt; FONT-FAMILY: Arial; mso-font-kerning: 0pt; mso-ansi-language: en" XML:LANG="EN">Duchenne</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">)肌营养不良症是此症最常见的形式,在美国有</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">1.3</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">万男童受其影响。因为它是由</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">X</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">染色体上一种基因缺陷引起,而女孩身上有该基因有第二拷贝,几乎从来没有女孩罹患此症。该新药,</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">eteplirsen</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">,针对</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">13</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">%由某种类型的基因突变所致肌肉营养不良症男童。如果不进行治疗,患者的肌肉不断衰弱,离不开轮椅,通常在</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">30</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">多岁匆匆离世。</SPAN></P> <P style="MARGIN: 0cm 0cm 0pt; TEXT-INDENT: 24pt; TEXT-ALIGN: left; mso-pagination: widow-orphan; mso-line-height-alt: 0pt; mso-char-indent-count: 2.0" align=left><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">如果</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">Eteplirsen</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">获得成功,这将是肌营养不良症患者和研究人员的巨大胜利,从未有任何药物或疗法可能使这些孩子得到丝毫好转。</SPAN></P> <P style="MARGIN: 0cm 0cm 0pt; TEXT-INDENT: 24pt; TEXT-ALIGN: left; mso-pagination: widow-orphan; mso-line-height-alt: 0pt; mso-char-indent-count: 2.0" align=left><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">获得这些结果也是</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">Sarepta</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">治疗药公司的一大胜利,坐落于美国马萨诸塞州剑桥这家生物技术公司开发该药。自从今年</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">7</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">月首次公布</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">12</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">名患者参与的一项研究数据之后,</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">Sarepta</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">的股价已经翻了超过</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">4</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">倍。数据显示</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">eteplirsen</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">增加一种关键的肌肉蛋白质——抗肌萎缩蛋白(</SPAN><SPAN lang=EN style="FONT-SIZE: 12pt; FONT-FAMILY: Arial; mso-font-kerning: 0pt; mso-ansi-language: en" XML:LANG="EN">dystrophin</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">)的量。在</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">8</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">月披露的</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">35</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">周结果显示:接受最大剂量该药的</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">4</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">名患者比服用安慰剂者在</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">6</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">分钟内再行走</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">69</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">米</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">。</SPAN></P> <P style="MARGIN: 0cm 0cm 0pt; TEXT-INDENT: 24pt; TEXT-ALIGN: left; mso-pagination: widow-orphan; mso-line-height-alt: 0pt; mso-char-indent-count: 2.0" align=left><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">现在,</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">48</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">周的数据显示这</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">4</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">名接受该药的男孩实际上比研究开始前多走</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">21</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">米</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">。在第</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">24</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">周让起初接受安慰剂的患者切换到服用</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">eteplirsen</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">。那些</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">“</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">延误治疗</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">”</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">的患者病情似乎也稳定了,</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">36</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">周的结果对他们进行的</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">6</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">分钟步行测试行走距离增加</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">10</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">米</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">,尽管比这一研究开始时仍然减少了</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">68</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">米</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">。接受最大剂量</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">eteplirsen</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">的患者比开始时使用安慰剂者能多走</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">89</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">米</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">,这一差异有统计学意义。</SPAN></P> <P style="MARGIN: 0cm 0cm 0pt; TEXT-INDENT: 24pt; TEXT-ALIGN: left; mso-pagination: widow-orphan; mso-line-height-alt: 0pt; mso-char-indent-count: 2.0" align=left><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">研究一开始就接受</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">eteplirsen</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">的患者抗肌萎缩蛋白检测呈阳性,他们的肌肉纤维数量增加了</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">47</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">%;先服用安慰剂,</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">24</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">周后切换为</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">eteplirsen</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">的患者肌肉纤维增加</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">38</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">%。</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US"><BR><FONT color=#333333><FONT face=Arial><SPAN style="mso-spacerun: yes">&nbsp;<WBR>&nbsp;<WBR>&nbsp;<WBR></SPAN> Sarepta</FONT></FONT></SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial; mso-hansi-font-family: Arial">公司的首</SPAN><SPAN lang=EN-US style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: Arial" XML:LANG="EN-US">??</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial">席执行官</SPAN><SPAN lang=EN style="FONT-SIZE: 12pt; FONT-FAMILY: Arial; mso-font-kerning: 0pt; mso-ansi-language: en" XML:LANG="EN">Chris Garabedian</SPAN><SPAN style="FONT-SIZE: 12pt; COLOR: #333333; FONT-FAMILY: 宋体; mso-bidi-font-family: Arial; mso-ascii-font-family: Arial">承认,这是新数据的最佳病例。</SPAN></P>
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