首页-庞贝氏罕见病关爱中心

<h5><a href="/article/e94550c93cd70fe748e6982b3439ad3b" target="_blank">清远患罕见病"庞贝氏症"女孩五年来首次回家(图)</a></h5>
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            <p> 2013年12月23日06:51  金羊网-羊城晚报郑宇宁近五年来第一次回家，妈妈给她封了利是羊城晚报记者周松摄本报关注下募70万善款病情有好转羊城晚报讯记者周松报道：广东地区唯一确诊的世界罕见病“庞贝氏症”患者郑宇宁，在社会各界的帮助下，用上了一种昂贵的特效药，经过将近一年的治疗，病情明显好转，22日冬至这天，郑宇宁近五年来第一次离开医院ICU病房，... </p>
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<h5><a href="/article/2e65f2f2fdaf6c699b223c61b1b5ab89" target="_blank">花季少女得罕见病--庞贝氏症靠呼吸机维持生命郑宇宁坚强面对</a></h5>
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            <p> http://v.youku.com/v_show/id_XNDQ0Nzk2OTc2.html </p>
            
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<h5><a href="/article/edfbe1afcf9246bb0d40eb4d8027d90f" target="_blank">帮庞贝氏症“奇迹女孩”延续奇迹</a></h5>
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            <p> 来源：天津北方网 作者：杨奕 2014-06-1615:02:17 编辑：寇庆春天津联通西青分公司党群工作部主管孙立强（中）接过爱心接力棒天津北方网讯：15岁，这本该是如花朵般绽放的年纪，本该享有着无忧无虑的青春时光、对未来充满着各种美好的憧憬。可对于张维，同样是15岁，这个女孩却要面对的是一个叫做“庞贝氏症”的世界罕见疾病。而在这条与病魔抗争的道路上... </p>
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<h5><a href="/article/df263d996281d984952c07998dc54358" target="_blank">青岛一幼童患罕见庞贝氏症 每月治疗费3.5万父母求助</a></h5>
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            <p> 来源:青岛电视台 作者: 我来说说 复制链接 2015-03-1209:15幼童患罕见庞贝氏症    这两天微博和微信朋友圈里，都在转一条九个月的宝宝得了罕见病急需治疗的帖子，记者联系上了孩子父母，见到了这个可怜的孩子。    9个月大的小果果，看上去和正常的孩子没什么两样，见到陌生人会害羞，见到记者采访的话筒会好奇，可爱的她现在还不明白，自己患上的究... </p>
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<h5><a href="/article/217eedd1ba8c592db97d0dbe54c7adfc" target="_blank">女婴患上庞贝氏症 急盼救命药</a></h5>
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            <p> 发布时间：2015年3月9日        果果看上去没有明显异样，实际亟待治疗。女孩果果刚出生9个月，当她度过人生的第一个春节时，还没学会叫妈妈的她被确诊为庞贝氏症。专家介绍，这是一种罕见病，如不及时治疗，果果面临的将是肌肉萎缩无力、心脏变大、呼吸能力逐渐衰竭，而多数患儿会因并发症在1岁前死亡。“感冒”原来是罕见病7日上午，家住李沧区的侯女士致电本报... </p>
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<h5><a href="/article/a5cdd4aa0048b187f7182f1b9ce7a6a7" target="_blank">基于国外的单臂小样本临床试验数据（注射用阿糖苷酶治疗庞贝病）</a></h5>
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            <p> 发布时间：2015-10-10来源：度衡之道庞贝病（PompeDisease）是一种由于溶酶体酸性α-葡萄糖苷酶（GAA）缺乏导致的溶酶体贮积症（LSD），又称酸性α-葡萄糖苷酶缺乏症或II型糖原贮积症(GlycogenstoragediseasetypeII,GSD-II)，以常染色体隐性方式遗传。庞贝病是一种罕见、进行性和致死性代谢性肌病，全球发病... </p>
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<h5><a href="/article/6bc24fc1ab650b25b4114e93a98f1eba" target="_blank">关注罕见病，增强庞贝病筛查意识，提高诊断效率——第四届亚洲庞贝病论坛报道</a></h5>
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            <p> 发布时间：2016-12-01来源：中国医学论坛报11月5~6日，第四届亚洲庞贝病论坛在上海召开，旨在帮助国内临床医生了解庞贝病（PD）研究进展，增强罕见病筛查意识，提高诊疗水平。华中科技大学同济医学院附属同济医院罗小平教授表示，本次会议有125位来自东亚和东南亚的代表，50%参会医生或学者来自海外。来自中国大陆、香港特别行政区、台湾，阿根廷，马来西亚... </p>
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<h5><a href="/article/6cd67d9b6f0150c77bda2eda01ae484c" target="_blank">庞贝氏病患儿治疗面临困局 要用一亿元续命三十年？</a></h5>
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            <p> 发布时间：2015-04-13来源：羊城晚报作者：记者：李国辉实习生：刘希曾国雄筹集到的钱只够小怡霖用药半年，但未来怎么办仍是个问号4月9日，被确诊为庞贝氏病的汕头2岁女婴曾怡霖，已在广州市儿童医院的ICU病房度过了近一个月时间，其间从未摘下呼吸机。为了救小怡霖，她的父母曾国雄夫妇辗转多日，已花14.9万余元在香港购买了24支治疗庞贝氏病唯一的特效药，... </p>
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<h5><a href="/article/e995f98d56967d946471af29d7bf99f1" target="_blank">FDA批准赛诺菲酶替代疗法Lumizyme用于全年龄段庞贝氏症（Pome）</a></h5>
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            <p> 发布时间：2014-08-06来源：生物谷赛诺菲（Sanofi）旗下健赞（Genzyme）7月31日宣布，FDA已批准扩大Lumizyme（α-葡萄糖苷酶，algucosidasealfa）适应症，用于所有年龄段或表型的庞贝氏症（Pompe）患者的治疗。此前，FDA于2010年5月仅批准Lumizyme用于8岁及以上晚发型庞贝氏症患者的治疗。庞贝氏症（... </p>
            
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<h5><a href="/article/6c29793a140a811d0c45ce03c1c93a28" target="_blank">庞贝病用药AT2220开始二阶段临床试验研究</a></h5>
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            <p> 发布时间：2012-02-02来源：中国医药123网    AmicusTherapeutics公司首次将AT2220（去氧野艽霉素盐酸盐）和酶代替治疗（ERT）注射液用于庞贝病（糖原累积症二型）患者。     多中心、开放式的二阶段研究010，通过AT2220和ERT共同治疗与ERT单一治疗作对比，目的是为了验证AT2220的安全性和药代动力学。  ... </p>
            
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<h5><a href="/article/ba3866600c3540f67c1e9575e213be0a" target="_blank">FDA批准首个针对四种遗传病的新生儿筛检系统—1型粘多糖病、庞贝氏症、高雪氏症和法布里病</a></h5>
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            <p> 发布时间：2017-02-10来源：药明康德    近日，美国FDA宣布批准新生儿筛检方案SeekerSystem，用于四种溶酶体贮积症（LysosomalStorageDisorders）的筛查。这是首个经FDA批准用于这些疾病筛查的方案。    溶酶体贮积症是一系列遗传性代谢疾病的总称。在正常人的体内，脂类、核酸、蛋白等不需要的代谢产物会被运往溶酶... </p>
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<h5><a href="/article/59c33016884a62116be975a9bb8257e3" target="_blank">川普举例庞贝氏症患者，号召FDA医药审批改革</a></h5>
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            <p> 发布时间：2017-04-28来源：中国罕见病    前不久美国的一次国会演讲中，唐纳德•川普总统介绍了MeganCrowley，一位罹患罕见病的20岁姑娘。    Crowley患有一种名为庞贝氏症（Pompedisease）的神经肌肉疾病。她将自己的存活，归功于父亲的小生物技术公司为她所患的罕见基因突变（疾病）开发和生产一种疗法。    演讲中，川... </p>
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<h5><a href="/article/d5cfead94f5350c12c322b5b664544c1" target="_blank">国外“孤儿药”入华提速：可有条件批准上市再补做研究</a></h5>
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            <p> 发布时间：2017-05-17来源：中国罕见病    美而赞，英文名称 Myozyme (Alglucosidasealfa, Lumizyme)，于2006年获得美国FDA首次批准，2010年5月美国FDA仅批准 Myozyme 用于8岁及以上晚发型庞贝氏症患者的治疗。2014年，FDA批准扩大Myozyme 适应症，用于所有年龄段或表型的庞贝氏症患... </p>
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<h5><a href="/article/5751ec3e9a4feab575962e78e006250d" target="_blank">小猕猴和庞贝氏的故事</a></h5>
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            <p> 小猕猴，出生于1980年初，女，在人生求学的路上渡过了悠悠23载，终于于09年进入社会，开始工作向着美好的生活迈进了。09年发生了很多事情，毕业了，工作了，结婚了，怀孕了，房奴了～ 10年小猕猴顺产生了宝宝。然而，这种平淡和美好却随着一纸体检书的到来破灭了。2011年9月，小猕猴在例行体检中发现谷丙转氨酶和谷草转氨酶均高于正常值（ALT：75，AST：... </p>
            
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<h5><a href="/article/d4c2e4a3297fe25a71d030b67eb83bfc" target="_blank">赛诺菲开始庞贝氏症第二代疗法3期临床试验</a></h5>
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            <p> 来源：中国罕见病网2017-01-1017:26            美国赛诺菲公司专业护理全球业务部门近日宣布：研究性疗法neoGAA的一项关键3期临床试验COMET已经招募到了首位患者并进行了一次输注。NeoGAA是一种针对庞贝氏症（Pompedisease）的第二代酶替代疗法。    庞贝氏症是一种进行性、使人衰弱致命的神经肌肉疾病，由溶酶体酶... </p>
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<h5><a href="/article/fb89705ae6d743bf1e848c206e16a1d7" target="_blank">国瑞：我是爸爸也是儿子</a></h5>
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            <p>     我叫国瑞，不要问我姓什么，因为我姓国。比较特殊的姓氏吧！我81年出生的，人们常说三十而立，然而我却在而立之时差点让连站都站不起来。2010年以前我原本生活的快快乐乐，顺利的上完大学，毕业、工作、结婚、生子，儿子聪明可爱，妻子温柔贤惠、父母身体健康，家庭和和睦睦。可这一切都因为一纸病例诊断给破坏的彻彻底底。自从2004年大学毕业以后，因为工作的原... </p>
            
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<h5><a href="/article/0d3180d672e08b4c5312dcdafdf6ef36" target="_blank">郑宇宁：明天的阳光都是恩赐</a></h5>
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            <p>     我叫郑宇宁，是广东省藉确诊的第一例罕见病“庞贝氏症”患者，也是清远市唯一的一例。19岁那年正向往着大学生活，却突发呼吸衰竭进ICU，今年25岁仍在ICU依靠呼吸机生存，板下手指都快6年了，小伙伴们都大学毕业闯荡江湖了，好羡慕啊！上大学----我的梦想啊！俺从小学开始，体育成绩就比同学们差一大截，俺还是挺求上进的，也不知是咋回事，好伤自尊。到了高... </p>
            
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<h5><a href="/article/82f2b308c3b01637c607ce05f52a2fed" target="_blank">李文华：真希望这只是一场梦</a></h5>
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            <p>     我叫李文华，家住山东省济宁市兖州大安镇李家宫村，家中兄弟姐妹五人，我排行第五。家里原本是一片和谐、幸福的氛围，但这样的平静却被无情的病魔摧毁。2011年6月份，当我在奋力拼搏备战研究生考试时，噩耗传来，与我龙凤胎的哥哥(李文峰)在广州重病住院，紧接着二姐(李静)由于压力过大，病情加重也被送入重症监护室治疗。烈日炎炎下，树叶被风吹得沙沙作响，而我... </p>
            
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<h5><a href="/article/788d986905533aba051261497ecffcbb" target="_blank">什么是庞贝氏症</a></h5>
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            <p>     庞贝氏症又称为酸性α-葡萄糖苷酶缺乏症或糖原贮积症Ⅱ型(GSDⅡ)。由于溶酶体内缺乏酸性α-葡萄糖苷酶(acidalpha-glucosidase,GAA)，使糖原及麦芽糖不能转化为葡萄糖而被利用，以致体内大量糖原在骨骼肌、心肌和平滑肌等组织细胞内聚积而致病。在北美及欧洲的发病率约为1:40,000，中国台湾发病率约为1:50,000。根据发病... </p>
            
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