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第九届国际杜氏会议
  发表日期:2012-08-31

9 th International Duchenne sponsored Conference

Over 300 delegates packed the meetings at this years Conference on November 4th and 5th in London. These are some of the presentations from this years Conference.

Learning and behaviour in Duchenne - Janet Hoskin Decipha

Janet explains how her Decipha programme has supported over 80 young people with Duchenne in schools across the UK. Supported by funding from the Big Lottery, Children in Need and Action Duchenne.

 

Learning and behaviour in Duchenne - Dr James Poysky USA

James is a practising psychologist in the US and also has a son with Duchenne. He explains the high risk of related learning and behaviour problems.

 

Stem Cells for Duchenne - Dr Jenny Morgan UCL and Professor Terry Partridge Washington USA

Jenny and Terry explain their research programmes that are trying to find ways of delivering stem cell treatments in Duchenne

 

Personalised Medicines for Duchenne - Dr Padraig Wright GSK

Padriag gives a detailed account of GSK's exon skipping programme and also explains the role of the newly formed rare disease unit at GSK

 

A review of Prosensa's exon skipping programme - Dr Giles Campion Netherlands

Giles describes Prosensa's extensive clinical trial programme to develop exon skipping drugs based upon their 2'OMe chemistry.

 

Enabling DMD clinical trials with large scale production of AAV vectors- Rob Kotin USA

Rob has developed with support from the NIH an important production facility in the US for AAV vectors. He describes here how vectors are produced and how these could be scaled up for future GMP prduction

 

Update on SMT1100 a utrophin upregulator for Duchenne - Jon Tinsley Summit Oxford

Jon and his team are now reworking the SMT1100 molecule for Duchenne. He describes recent progress at biopharma Summit.

 

AAV based mRNA therapies for Duchenne - Dr Luis Garcia Paris France

Luis has been working towards a clinical trial using AAV vectors that carry skipping promoter. He describes in detail how this system might work in humans and animal models and progress so far.

 

Expectations of Clinical trials - Susie Darricott Prosensa Netherlands

Susie from the biopharma Prosensa discusses the process of clincal trials.

 

Discovering Drugs for Duchenne - Dr Ellen Welch PTC Therapuetics USA

Ellen highlights PTC's drug discovery programme for Duchenne including Ataluren

 

Biglycan as a therapy for Duchenne - Dr Justin Fallon USA

Justin discusses the use of Biglycan for Duchenne

 

Physiotherapy - Marion Main Great Ormond Street London

Marion describes the need for physiotherapy to support those living with Duchenne.

 

Targeting therapuetic molecules in the mitochondria - Dr Mike Murphy Cambridge

Mike has created a drug called MitoQ that potentially acts as a powerful antioxidant by being targeted at the Mitochondria. Would this be of benefit in Duchenne

 

Light at the end of the tunnel and the debate between Nick Catlin and Annemeike Rus on N=1 clinical trials

Kay Davies updates us on the recent developments in Duchenne research as well as highlighting progress in her labs Utrophin up-regulation programme.
Both sides of the debate about the problems of present trial protocols in Duchenne is presented by Nick and Annemeike. Nick gives an alternative N=1 protocol.

 

Early Treatment with Lisinopril and spironolactone for the heart - Paul Jansen USA

Paul's team have recently completed a study on the ACE inhibitor Lisinopril and he reports here some promising results.

 

IGF-1 therapy and muscle function in Duchenne - Dr Meilan Rutter USA

Meilan and the team in Cincinnati Children's Hospital have been recruiting for a trial of IGF-1. This is also part of the Pilot Trials Now programme supported by Action Duchenne.

 

Catena for Duchenne - Dr Nicholas Coppard Santhera Switzerland

Nicholas has presented the results of the trials on Santhera's Catena drug for patients in Duchenne Muscular Dystrophy

 

Revatio for heart disease in DBMD - Dr Dan Judge USA

Dan and his team have been recruiting patients for a trial on sildenifil or viagra to see if this stabilises problems with the heart in Duchenne. This is part of the Pilot Trials Now programme supported by Action Duchenne.

 

The Holy Grail of Multiple exon skipping - Professor Terry Partridge Washington USA

Terry maps out strategies for multi skipping that will be necessary if we are able to treat a the widest range of those living with Duchenne with exon skipping drugs.

 

Action of ACE inhibitor perindopril on cardio skeletal muscle in Duchenne - Dr Denis Duboc France

Denis has nearly completed an extensive clinical trial using Perindopril. he discusses the potential of this drug for delaying heart symptoms and also the possible effects on skeletal muscle.

 

Peptide based inhibition of NF-kB - Professor Paul Jansen USA

Paul discusses his teams published research into down regulating NF-kB as a potential therapy for Duchenne.

 

AVI 4658 Clinical Trials - Dr Ed Kaye AVI Biopharma

Ed explains the development of AVI's exon skipping drug and the results of clinical trials

 

Endocrinology in Duchenne - Dr Meilan Rutter USA

Meilan explains the role of the endocrine system in the human body and how this can be disrupted by the long term use of steroids.

 

MDEX Clinical Trial Programme and study 28 - Professor Francesco Muntoni - UCL London

Francesco explains the successful completion of the MDEX clinical trials using AVI's morpholino exon skipping drugs. He also maps out further MDEX trials and programmes.

 

Bone Protection for children with Duchenne treated with corticosteroids - Dr Ros Quinliven Great Ormond Street London

Steroids are recommended as a key drug that helps to slow the muscle wasting in Duchenne. However there are a number of side effects that can be experienced when taking steroids over a long period. Here Ros explains the cause and how best to manage these problems. Action Duchenne has produced a booklet edited by Ros that is available from our Office.

 

Using AAV vectors and microdystrophin Professor George Dickson Royal Holloway London

By using AAV vectors to package a gene to replace the faulty dystrophin George's gene therapy aims to provide a body wide treatment that could slow the muscle wasting for all boys. Action Duchenne is also funding a new and exciting AAV project in the Royal Holloway lab.

 

Duchenne Alliance - Christine McSherry Jett Foundation USA

Action Duchenne has always supported collaboration with other Duchenne non profits. Christine has recently set up the Duchenne Alliance to bring together Duchenne funders so that we can all focus our efforts internationally on new drugs and treatments for Duchenne. The Alliance has just launched the Duchenne Dashboard a unique online platform that offers researchers and funders the chance to review and find backing for new projects.

 

Improving the design of antisense drugs using peptides - Professor Matthew Wood Oxford

Matthew in collaboration with Dr Mike Gait at Cambridge has been improving the design of the chemistry of drugs that make exon skipping work. The new peptide conjugates of antisense oligonucleotides are now being developed for clinical trial.

 

Improving the design of antisense drugs using peptides - Dr Mike Gait Cambridge

Mike in collaboration with Professor Matthew Wood at Oxford has been improving the design of the chemistry of drugs that make exon skipping work. The new peptide conjugates of antisense oligonucleotides are now being developed for clinical trial.

 

March of the Young men - Gordon McClurg

Gordon McClurg was diagnosed with Duchenne at the age of 3. He now runs Care Management Services that supports people with a variety of neuromuscular conditions that want to live independently.

 

Introduction to exon skipping - Professor Steve Wilton Australia

Steve takes you through an excellent description of exon skipping and the problems with the dystrophin gene that are cause of Duchenne Muscular Dystrophy. He also updates us all on recent developments in new drugs for exon skipping. Listen and look at the slides from the presentation.

 

Caring for the Heart - Dr John Bourke Newcastle

John outlines the problems associated with the Heart and Duchenne. He describes how the early use of ACE inhibitors and Beta Blockers can help to maintain heart function for longer. There is news of his "Heart protection Trial" currently undergoing recruitment.

 

Biomarkers for Clinical Trials - Dr Sebahattin Cirak UCL London

The aim of this study is to identify modifier genes for Duchenne that can be used to monitor disease progress and drug response that could become surrogate markers for clinical trials.

 

What do NHS Reforms mean for Patient Care? Changing the map for Duchenne

Eilidh Macpherson from Action Duchenne, Arlene Wilkie Neurological Alliance and Alastair Kent discuss the current Government reforms to the NHS and its impact on services for Duchenne.

 

There is an Elephant in the Room - Progress of Duchenne Research

Dr Karl Bettelheim who is a Trustee for Action Duchenne and retired Scientific Researcher explores the widespread use of supplements and devices readily available on the internet.

在2011年的114日和5日在伦敦会议的300多名代表参加。这些都是一些介绍,从今年的会议。

学习和行为的杜氏 珍妮特·霍斯金Decipha

珍妮特解释说,她Decipha程序已经支持超过80个年轻人在英国各地的学校与杜氏。从大乐透的资金支持,儿童的需求和行动杜氏。

杜氏 詹姆斯博士Poysky美国的学习和行为

詹姆斯是一个在美国执业心理学家和杜氏进行性也有一个儿子。他解释相关的学习和行为问题的高风险。

干细胞杜氏 珍妮摩根博士伦敦大学学院和教授特里·帕特里奇华盛顿美国

珍妮和特里解释自己的研究计划,正在努力寻找方式提供干细胞治疗假肥大型

个性化的杜氏 哈灵顿莱特博士葛兰素史克公司的药品

Padriag给出了详细的帐户葛兰素史克(GSK)的外显子跳跃程序,也说明了罕见的疾病,新成立的单位在葛兰素史克的作用

贾尔斯坎皮恩博士荷兰回顾外显子跳跃程序 

,贾尔斯描述广泛的临床试验计划,发展外显子跳跃根据后2'OMe化学药物的。

启动的DMD临床试验与大规模生产的腺相关病毒载体罗布Kotin美国

罗布从美国国立卫生研究院的一个重要生产设施在美国的AAV载体的支持。他描述了他在这里载体是如何产生的,以及如何可以扩大这些未来的GMP涂璇,薛泉宏,司美茹,龚明福

更新SMT1100一个杜氏 乔恩·廷斯利首脑会议上牛津大学的utrophin upregulator

乔恩和他的团队正在改造SMT1100分子杜氏。他介绍了在生物制药峰会的最新进展。

杜氏腺相关病毒为基础的基因疗法 路易斯·加西亚博士法国巴黎

,路易斯一直致力的临床试验用腺相关病毒载体携带。他介绍,这个系统是如何工作的细节至今在人类和动物模型和进步。

预期的临床试验 苏西Darricott Prosensa荷兰

苏西的生物制药Prosensa的讨论的过程中,clincal试验。

发现药物杜氏 艾伦·韦尔奇博士美国PTC Therapuetics

在包括Ataluren,艾伦凸显PTC的药物发现计划,杜氏

聚糖作为一种治疗Duchenne型 美国博士贾斯汀伦

贾斯汀讨论了使用聚糖杜氏

物理治疗 马里昂主伦敦大奥蒙德街

马里昂物理治疗师的描述,以支持这些进行性杜氏生活的需要。

针对therapuetic分子中的线粒体 迈克墨菲剑桥博士

迈克创建了一个的药物称为MitoQ可能作为一个强大的抗氧化剂,通过有针对性的线粒体。这有利于在杜氏

在隧道尽头的光,尼克卡特林和Annemeike的罗斯之间的争论N = 1的临床试验

凯戴维斯更新,我们在杜氏研究的最新发展,以及突出的进步,在她的实验室Utrophin上调方案。 
双方的辩论有关的问题,目前的试验协议,在杜氏由尼克和Annemeike的。尼克给出了另一种N = 1协议。

赖诺普利和安体舒通对心脏的早期治疗 保罗·詹森美国

保罗的团队最近完成的一项研究对ACE抑制剂赖诺普利和他的报告在一些可喜的成果。

IGF-1治疗和肌肉功能杜氏 美兰鲁特美国博士

美兰在美国辛辛那提儿童医院的团队已经招募的IGF-1的试验。这也是现在的试点试验程序所支持的行动杜氏的一部分。

系列杜氏 尼古拉斯科珀德博士Santhera瑞士

杜氏肌营养不良症的患者,尼古拉斯提出Santhera的卡泰纳药物试验的结果

Revatio治疗心脏疾病DBMD - 美国博士丹法官

丹和他的团队已经招募患者的试验在sildenifil或伟哥,看看是否稳定在杜氏与心脏问题。这是现在的试点试验程序所支持的行动杜氏的一部分。

圣杯的多外显子跳跃 教授特里·帕特里奇华盛顿美国

特里映射出多跳过这将是必要的,如果我们是能治疗那些生活范围最广的与Duchenne型外显子跳跃药物的战略。

行动的ACE抑制剂培哚普利对心骨骼肌杜氏 医生丹尼斯Duboc法国

丹尼斯已经接近完成了一项广泛的临床试验,使用培哚普利。他讨论了这种药物潜在的延迟心脏病的症状,并可能产生的影响对骨骼肌。

肽为基础的抑制NF-kB的 美国教授保罗·詹森

保罗讨论他的团队发布的研究下调NF-KB作为一个潜在的治疗Duchenne型。

AVI 4658版凯博士临床试验 - AVI生物制药

埃德解释AVI外显子跳跃药物的开发和临床试验结果

内分泌杜氏 美兰鲁特美国博士

美兰解释在人体的内分泌系统的作用,以及如何可以打乱了的长期使用的类固醇类。

MDEX临床试验计划及研究28 - 的弗朗西斯Muntoni教授 - UCL伦敦

弗朗西斯解释MDEX使用AVI的吗啉外显子跳跃药物的临床试验的顺利。他还进一步描绘了MDEX试验和方案。

罗斯Quinliven博士伦敦大奥蒙德街儿童用皮质激素治疗假肥大型骨保护

类固醇被推荐为关键的药物,有助于减缓在杜氏肌肉萎缩。然而也有一些副作用,可以服用类固醇时,在很长一段经历。在这里,罗斯解释的原因和如何最好地处理这些问题。杜氏行动产生的活性氧,可以从我们的办公室编辑了一本小册子。

使用腺相关病毒载体和microdystrophin教授乔治·迪克森伦敦皇家霍洛威

通过使用打包基因的AAV载体,以取代出现故障的抗肌萎缩蛋白乔治的基因治疗的目的是,提供一个的身体广泛的治疗,可以减缓肌肉萎缩和所有的男孩。行动,杜氏还资助一个新的和令人兴奋的的AAV项目在皇家霍洛威实验室的。

杜氏联盟 McSherry杰特基金会美国

行动杜氏一贯支持与其他杜氏非利润的合作。克里斯蒂娜最近成立的的杜氏联盟汇集杜氏资助者,这样我们就可以集中我们的努力,在国际上新的药物和治疗Duchenne型。该联盟已杜氏仪表板刚刚推出的一个独特的网上平台,提供研究人员和资助者的机会,检讨及寻找新项目的支持。

改进设计的反义药物的使用肽 牛津大学教授马修伍德

马修博士在剑桥大学的迈克步态合作在不断提高设计的化学药物,使外显子跳跃的工作。反义寡核苷酸的新的肽结合物的目前正在开发用于临床试验。

改进设计的反义药物的使用肽 迈克步态剑桥博士

迈克在与马修伍德教授在牛津大学合作在不断提高设计的化学药物,使外显子跳跃的工作。反义寡核苷酸的新的肽结合物的目前正在开发用于临床试验。

3月的年轻男子 戈登麦克勒格

戈登麦克勒格被诊断出患有杜氏在3岁以下。他现在运行保障管理服务,支持与多种神经肌肉要独立生活的条件。

外显子跳跃 澳大利亚​​教授史蒂夫威尔顿

史蒂夫带你穿越一个很好的说明外显子跳跃的抗肌萎缩蛋白基因的问题是杜氏肌营养不良症的原因。他还更新我们最近的事态发展,新的药物外显子跳跃。听和看的幻灯片演示文稿。

关爱之心 约翰·伯克博士纽卡斯尔

约翰的心和杜氏列出了相关的问题。他介绍了如何早期使用ACE抑制剂和β受体阻滞剂可以帮助维持心脏功能更长的时间。有消息称,他的心脏保护试验,目前正在进行招聘。

临床试验的生物标志物 - Sebahattin Cirak博士UCL伦敦

本研究的目的是要找出Duchenne型的修饰基因,可以用来监测疾病的进展和对药物的反应,可能会成为临床试验的替代标记物。

NHS改革意味着什么病人的护理呢?改变杜氏地图

Eilidh行动杜氏,阿琳威尔基神经病学联盟和阿拉斯泰尔·肯特的麦花臣,讨论目前的政府改革的NHS服务为杜氏及其影响的。

有一个房间里的大象 杜氏研究进展

谁是受托人行动杜氏科学研究员和退休的医生卡尔贝特尔海姆探讨很容易在互联网上提供的补充和设备的广泛使用。

 
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