blue liquidity Sickle Cell Disease - Pipeline Review, H2 2014, 鐮形血球貧血症的開發中產品分析, 镰刀形红血球, 除生殖细胞和没有细胞核的红血球和血小板外，人体内所有的细胞（叫体细胞，以与生殖细胞相区别）都含有完全相同的DNA

另一种情况不是细胞衰亡，而是细胞变异。比如镰刀形红血球贫血症。由于血红蛋白上一个氨基酸单位的变化，红血球虽然生成了，却不能有效地输送氧气。更坏的情况是细胞癌变。细胞不但不死，还无节制地大量增殖。从癌症发生原因的最新观点来看，是在干细胞阶段发生了突变。细胞不能按正常的途径分化成熟，而是形成大量未充分分化的细胞。一个最明显的例子就是白血病，血液里有大量未充分分化的白细胞。所以肿瘤也有自己的干细胞，叫癌症干细胞，只有它们才能在肿瘤扩散中起作用，在身体的其它地方落脚生根，发展出新的肿瘤。并且它们不怕化疗。这是化疗能杀死大部分的肿瘤细胞，却不能根治肿瘤的原因。在这些情况下，变化了的干细胞本身就是疾病之源。     对于丧失功能的细胞，比如镰刀形红血球，可以在人体内引入正常的造血干细胞，或从患者的骨髓中取出造血干细胞，放入正常的血红蛋白基因，再输回患者体内，以生成功能正常的红血球。对于白血病，也可以用正常骨髓的干细胞来取代患者的骨髓，生产出正常的白细胞。对于其它肿瘤，就要找到消灭它们的癌症干细胞的办法，比如激活它们的程序控制自杀机制（programmed cell death，或称 apoptosis），或找到阻止这些癌症干细胞生成新细胞的方法，从源头上消灭肿瘤。   市場調查報告書 - 213124 血清蛋白是衡量血样中蛋白质总量的一个标准。健康者的人体血样大约由70%的蛋白质组成，它们在调节凝血和提供免疫力等方面扮演着重要角色。血清蛋白改变有可能是存在健康问题的迹象，因此可以给医生提供重要的诊断信息。 在血清蛋白检查中，会从患者体内抽取血样。为了更好的测试，可能会用多个试管装血。对抽血害怕的人最好是询问医生了解基本情况，并可以考虑在抽血前后吃些小吃缓解压力。抽出的血样会送到实验室进行化验。 确定血清蛋白数量有可能技术可以选择。血液中有球蛋白和白蛋白两种蛋白质，它们构成血液蛋白质的大部分，并且比例也保持相对稳定。如果这两种蛋白质的比例发生变化，往往与存在健康问题有关。可以导致血清蛋白变化的疾病包括脱水，糖尿病，心力衰竭，肾脏病，肺结核，肝脏疾病，自身免疫性疾病和白血病等血液疾病。 正常血清蛋白是每十升6到8克。如果发现这个值异常，还需要额外做检查找出原因。在诊断时，医生还会将其他检查结果考虑在内，如肝脏疾病患者的肝酶水平异常等。此外，医生还会把血清蛋白检查作为常规体验的一部分，观察对特定治疗的反应情况 鐮形血球貧血症的開發中產品分析 Sickle Cell Disease - Pipeline Review, H2 2014 出版商 Global Markets Direct 出版日期 2014年10月31日 內容資訊 英文 164 Pages 價格 USD 2,000 PDF by E-mail (Single user license) USD 4,000 PDF by E-mail (Site license) USD 6,000 PDF by E-mail (Global license) 鐮形血球貧血症的開發中產品分析 Sickle Cell Disease - Pipeline Review, H2 2014 出版日期: 2014年10月31日 內容資訊: 英文 164 Pages

簡介 鐮狀細胞貧血症為遺傳性(先天性)的血液疾病，血液中的紅血球形狀異常。主要的前兆、症狀為貧血和發育遲緩、視野障礙、疼痛、頻繁感染感染疾病等。主要的治療方法，有抗生素和止痛藥、輸血、幹細胞移植等。 本報告提供全球各國的鐮形血球貧血症治療用的開發中產品的開發情形相關分析、產品開發與上市的最新趨勢，及臨床實驗各階段的產品一覽，再加上主要企業簡介、主要藥物概要、最新的產業趨勢等資訊。 目錄 簡介 分析範圍 鐮形血球貧血症概要 治療藥的開發 鐮形血球貧血症的開發中產品:概要 鐮形血球貧血症的開發中產品:比較分析 各企業開發中的鐮形血球貧血症治療藥 大學/研究機關研究中的鐮形血球貧血症治療藥 開發中產品的概要 後期階段的產品 臨床實驗階段的產品 初期階段的產品 不明確階段的產品 鐮形血球貧血症治療藥:開發中產品一覽(各企業) 鐮形血球貧血症治療藥:研究中的產品一覽(各大學/研究機關) 鐮形血球貧血症治療藥的開發企業 Before the financial crisis, dealer market-making held a powerful place in the nation’s biggest investment banks. The fresh-out-of-college traders that shuffled in and out of Manhattan bank buildings each day handled the banks' inventories of bonds worth hundreds of millions of dollars, which they used as starting capital to make their own trades, says Lawrence McDonald, a former Lehman Bros. corporate bond trader. The holdings, which were used to make markets for each type of corporate security, would fit into two categories, or books. “The front book is the facilitation book. That’s the book for client liquidity. The back book is your prop position. That book was responsible for a lot of profits on the Street in 2007, and that today pretty much doesn’t exist,” said McDonald, who authored A Colossal Failure of Common Sense, a bestseller about the bank's demise. That system crumbled in the years after the financial crisis. The facilitation books shrank in size while the proprietary trading books became virtually non-existent. Dealer balance sheet sizes fell to a fraction of what they once were. The smaller amount that dealers hold on their books means they can’t unconditionally buy bonds that investors are trying to sell, especially in large blocks Johnson & Johnson 第一三共 Sangamo BioSciences, Inc. Gamida Cell Ltd. Novartis AG Astellas Pharma Inc. Addex Therapeutics Mast Therapeutics, Inc. Alnylam Pharmaceuticals, Inc. Cellerant Therapeutics, Inc. Advinus Therapeutics Ltd. Prolong Pharmaceuticals SynZyme Technologies, LLC Erytech Pharma SA Archemix Corp. Morphogenesis, Inc. NKT Therapeutics, Inc. Acetylon Pharmaceuticals, Inc. Emmaus Medical, Inc. AesRx, LLC Errant Gene Therapeutics, LLC Dilaforette AB OrphageniX, Inc. ReveraGen BioPharma, Inc. 鐮形血球貧血症:治療藥的評估 單劑治療藥的情況 並用治療藥的情況 各標的 各作用機制 各投藥法 各分子類型 藥物簡介 prasugrel hydrochloride 產品概要 功能機制 研究開發(R&D)的發展情形 麩醯胺酸 硫酸鎂 vepoloxamer Aes-103 regadenoson montelukast sodium+羥基尿素 LentiGlobin NiCord panobinosutatto zileuton CR(調整放出) sevuparin sodium Sanguinate tetrahydrouridine+decitabine NKTT-120 CNTO-530 Enhoxy Trichostatin A 鐮形血球貧血症用P-Selectin抑制用核酸適體 異常血色素症用ZFP轉錄因子 鐮形血球貧血症用DNA寡核苷酸 VBP-15 PNQ-103 鐮形血球貧血症用遺傳基因療法 ALN-TMP Lentiviral-Mediated遺傳基因療法 AIC-6020 ACY-957 VitalHeme EdX-17 AIC-2011 非惡性血液疾病用中央記憶T細胞 HbF誘導物質計劃 Minihepcidins SCPF 小分子TNFR1-NAM計劃 MSKCC GTx011 CLT-001 鐮形血球貧血症治療藥:開發中產品的最新趨勢 鐮形血球貧血症治療藥:暫停開發的產品 鐮形血球貧血症治療藥:開發中止的產品 鐮形血球貧血症相關產品的開發里程碑 值得注意的最新趨勢、新聞稿(共10件) 附錄 圖表一覽 ※ 本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。 目錄 Summary Global Markets Direct's, 'Sickle Cell Disease - Pipeline Review, H2 2014', provides an overview of the Sickle Cell Disease's therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Sickle Cell Disease, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Sickle Cell Disease and special features on late-stage and discontinued projects. Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct's proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis. The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease. Scope The report provides a snapshot of the global therapeutic landscape of Sickle Cell Disease The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities The report reviews key players involved in the therapeutics development for Sickle Cell Disease and enlists all their major and minor projects The report summarizes all the dormant and discontinued pipeline projects A review of the Sickle Cell Disease products under development by companies and universities/research institutes based on information derived from company and industry-specific sources Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages A detailed assessment of monotherapy and combination therapy pipeline projects Coverage of the Sickle Cell Disease pipeline on the basis of target, MoA, route of administration and molecule type Latest news and deals relating related to pipeline products Reasons to buy Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage Develop strategic initiatives by understanding the focus areas of leading companies Identify and understand important and diverse types of therapeutics under development for Sickle Cell Disease Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline Devise corrective measures for pipeline projects by understanding Sickle Cell Disease pipeline depth and focus of Indication therapeutics Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline Table of Contents Table of Contents List of Tables List of Figures Introduction Global Markets Direct Report Coverage Sickle Cell Disease Overview Therapeutics Development Pipeline Products for Sickle Cell Disease - Overview Pipeline Products for Sickle Cell Disease - Comparative Analysis Sickle Cell Disease - Therapeutics under Development by Companies Sickle Cell Disease - Therapeutics under Investigation by Universities/Institutes Sickle Cell Disease - Pipeline Products Glance Late Stage Products Clinical Stage Products Early Stage Products Sickle Cell Disease - Products under Development by Companies Sickle Cell Disease - Products under Investigation by Universities/Institutes Sickle Cell Disease - Companies Involved in Therapeutics Development Acceleron Pharma, Inc. Acetylon Pharmaceuticals, Inc. Addex Therapeutics Ltd Advinus Therapeutics Ltd. AesRx, LLC. Alnylam Pharmaceuticals, Inc. Archemix Corp. Bluebird bio, Inc. Bristol-Myers Squibb Company Children's Hospital Boston Daiichi Sankyo Company, Limited Dilaforette AB Emmaus Medical, Inc. Errant Gene Therapeutics, LLC Erytech Pharma SA Gamida Cell Ltd. Gilead Sciences, Inc. Global Blood Therapeutics, Inc. Isis Pharmaceuticals, Inc. Johnson & Johnson Mast Therapeutics, Inc. Morphogenesis, Inc. NKT Therapeutics, Inc. Novartis AG OrphageniX, Inc. Pfizer Inc. Prolong Pharmaceuticals ReveraGen BioPharma, Inc. Sangamo BioSciences, Inc. Sanofi SynZyme Technologies, LLC Sickle Cell Disease - Therapeutics Assessment Assessment by Monotherapy Products Assessment by Target Assessment by Mechanism of Action Assessment by Route of Administration Assessment by Molecule Type Drug Profiles ACY-957 - Drug Profile ADX-85142 - Drug Profile ADX-94819 - Drug Profile Aes-103 - Drug Profile AIC-2011 - Drug Profile AIC-6020 - Drug Profile ALN-TMP - Drug Profile Antisense Oligonucleotide to Inhibit BCL11A and KLF1 for Hematological Disorders - Drug Profile apixaban - Drug Profile ARC-5690 - Drug Profile Cell Therapy for Sickle Cell Disease - Drug Profile CNTO-530 - Drug Profile DNA Oligonucleotides for Sickle Cell Anemia - Drug Profile Drugs to Inhibit PRMT5 for Sickle Cell Anemia - Drug Profile EdX-17 - Drug Profile Enhoxy - Drug Profile Gene Therapy for Hemoglobinopathies - Drug Profile Gene Therapy for Sickle Cell Anemia - Drug Profile Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile Gene Therapy to Activate Gamma Globin for Sickle Cell Disease - Drug Profile glutamine - Drug Profile GTX-011 - Drug Profile LentiGlobin - Drug Profile luspatercept - Drug Profile NiCord - Drug Profile NKTT-120 - Drug Profile panobinostat - Drug Profile PB-04 - Drug Profile PF-4447943 - Drug Profile plerixafor - Drug Profile PNQ-103 - Drug Profile prasugrel hydrochloride - Drug Profile regadenoson - Drug Profile RN-1 - Drug Profile Sanguinate - Drug Profile SCPF - Drug Profile sevuparin sodium - Drug Profile Small Molecule to Agonize A2A for Immunology, Hematological and Neurological Disorders - Drug Profile Small Molecules to Inhibit TNFR1 for Immunology, Gastrointestinal, Neurology and Hematological Disorders - Drug Profile Stem Cell Therapy to Activate Beta Globin for Sickle Cell Disease - Drug Profile Synthetic Peptides Hematological and Genetic Disorders - Drug Profile Trichostatin A - Drug Profile VBP-15 - Drug Profile vepoloxamer - Drug Profile VitalHeme - Drug Profile Sickle Cell Disease - Recent Pipeline Updates Sickle Cell Disease - Dormant Projects Sickle Cell Disease - Discontinued Products Sickle Cell Disease - Product Development Milestones Featured News & Press Releases Appendix Methodology Coverage Secondary Research Primary Research Expert Panel Validation Contact Us Disclaimer List of Tables Number of Products under Development for Sickle Cell Disease, H2 2014 Number of Products under Development for Sickle Cell Disease - Comparative Analysis, H2 2014 Number of Products under Development by Companies, H2 2014 Number of Products under Investigation by Universities/Institutes, H2 2014 Comparative Analysis by Late Stage Development, H2 2014 Comparative Analysis by Clinical Stage Development, H2 2014 Comparative Analysis by Early Stage Development, H2 2014 Products under Development by Companies, H2 2014 Products under Development by Companies, H2 2014 (Contd..1) Products under Development by Companies, H2 2014 (Contd..2) Products under Investigation by Universities/Institutes, H2 2014 Sickle Cell Disease - Pipeline by Acceleron Pharma, Inc., H2 2014 Sickle Cell Disease - Pipeline by Acetylon Pharmaceuticals, Inc., H2 2014 Sickle Cell Disease - Pipeline by Addex Therapeutics Ltd, H2 2014 Sickle Cell Disease - Pipeline by Advinus Therapeutics Ltd., H2 2014 Sickle Cell Disease - Pipeline by AesRx, LLC., H2 2014 Sickle Cell Disease - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2014 Sickle Cell Disease - Pipeline by Archemix Corp., H2 2014 Sickle Cell Disease - Pipeline by Bluebird bio, Inc., H2 2014 Sickle Cell Disease - Pipeline by Bristol-Myers Squibb Company, H2 2014 Sickle Cell Disease - Pipeline by Children's Hospital Boston, H2 2014 Sickle Cell Disease - Pipeline by Daiichi Sankyo Company, Limited, H2 2014 Sickle Cell Disease - Pipeline by Dilaforette AB, H2 2014 Sickle Cell Disease - Pipeline by Emmaus Medical, Inc., H2 2014 Sickle Cell Disease - Pipeline by Errant Gene Therapeutics, LLC, H2 2014 Sickle Cell Disease - Pipeline by Erytech Pharma SA, H2 2014 Sickle Cell Disease - Pipeline by Gamida Cell Ltd., H2 2014 Sickle Cell Disease - Pipeline by Gilead Sciences, Inc., H2 2014 Sickle Cell Disease - Pipeline by Global Blood Therapeutics, Inc., H2 2014 Sickle Cell Disease - Pipeline by Isis Pharmaceuticals, Inc., H2 2014 Sickle Cell Disease - Pipeline by Johnson & Johnson, H2 2014 Sickle Cell Disease - Pipeline by Mast Therapeutics, Inc., H2 2014 Sickle Cell Disease - Pipeline by Morphogenesis, Inc., H2 2014 Sickle Cell Disease - Pipeline by NKT Therapeutics, Inc., H2 2014 Sickle Cell Disease - Pipeline by Novartis AG, H2 2014 Sickle Cell Disease - Pipeline by OrphageniX, Inc., H2 2014 Sickle Cell Disease - Pipeline by Pfizer Inc., H2 2014 Sickle Cell Disease - Pipeline by Prolong Pharmaceuticals, H2 2014 Sickle Cell Disease - Pipeline by ReveraGen BioPharma, Inc., H2 2014 Sickle Cell Disease - Pipeline by Sangamo BioSciences, Inc., H2 2014 Sickle Cell Disease - Pipeline by Sanofi, H2 2014 Sickle Cell Disease - Pipeline by SynZyme Technologies, LLC, H2 2014 Assessment by Monotherapy Products, H2 2014 Number of Products by Stage and Target, H2 2014 Number of Products by Stage and Mechanism of Action, H2 2014 Number of Products by Stage and Route of Administration, H2 2014 Number of Products by Stage and Molecule Type, H2 2014 Sickle Cell Disease Therapeutics - Recent Pipeline Updates, H2 2014 Sickle Cell Disease - Dormant Projects, H2 2014 Sickle Cell Disease - Discontinued Products, H2 2014 List of Figures Number of Products under Development for Sickle Cell Disease, H2 2014 Number of Products under Development for Sickle Cell Disease - Comparative Analysis, H2 2014 Number of Products under Development by Companies, H2 2014 Number of Products under Investigation by Universities/Institutes, H2 2014 Comparative Analysis by Clinical Stage Development, H2 2014 Comparative Analysis by Early Stage Products, H2 2014 Assessment by Monotherapy Products, H2 2014 Number of Products by Top 10 Targets, H2 2014 Number of Products by Stage and Top 10 Targets, H2 2014 Number of Products by Top 10 Mechanism of Actions, H2 2014 Number of Products by Stage and Top 10 Mechanism of Actions, H2 2014 Number of Products by Top 10 Routes of Administration, H2 2014 Number of Products by Stage and Top 10 Routes of Administration, H2 2014 Number of Products by Top 10 Molecule Types, H2 2014 Number of Products by Stage and Top 10 Molecule Types, H2 2014