更新于：2024-11-01

Von Willebrand Diseases

冯·维勒布兰德病

更新于：2024-11-01

基本信息

别名

ANGIOHEMOPHILIA、Angiohaemophilia、Angiohemophilia

+ [100]

简介

Group of hemorrhagic disorders in which the VON WILLEBRAND FACTOR is either quantitatively or qualitatively abnormal. They are usually inherited as an autosomal dominant trait though rare kindreds are autosomal recessive. Symptoms vary depending on severity and disease type but may include prolonged bleeding time, deficiency of factor VIII, and impaired platelet adhesion.

关联

项与冯·维勒布兰德病相关的药物

Efanesoctocog alfa

靶点

F10

作用机制

F10刺激剂

在研机构

赛诺菲（中国）投资有限公司 [+6]

原研机构

Amunix, Inc.

在研适应症

血友病A [+1]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2023-02-22

Emicizumab

艾美赛珠单抗

靶点

F10 x factor IXa

作用机制

F10抑制剂 [+1]

在研机构

Hoffmann-La Roche, Inc. [+9]

原研机构

Chugai Pharmaceutical Co., Ltd.

在研适应症

凝血因子缺乏症 [+3]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2017-11-16

Vonicog Alfa (Baxalta)

伏尼凝血素α

靶点

vWF

作用机制

vWF刺激剂

在研机构

Baxalta, Inc. [+6]

原研机构

Baxalta, Inc.

在研适应症

冯·维勒布兰德病 [+2]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2015-12-08

152

项与冯·维勒布兰德病相关的临床试验

NCT06651255 / Not yet recruitingN/AIIT

Algorithm-based Management to Reduce the Recurrence of Gastrointestinal Bleeding and Severe Epistaxis in Von Willebrand Disease: WILL MANAGE Trial

WILL MANAGE is a prospective multicenter controlled open label randomized trial comparing an algorithm-based multidisciplinary management to a standard of care to reduce the incidence of bleeding (GI bleeding or severe epistaxis) recurrence in von Willebrand disease.

开始日期2025-01-06

申办/合作机构-

NCT06610201 / RecruitingN/A

A Prospective, Screening Study of Bleeding and Treatment in Participants With Von Willebrand Disease

The purpose of this screening study is to accumulate information regarding bleeding events, quality of life, and the social and clinical impact of bleeds in participants with Von Willebrand Disease (VWD). Data from this study will be used to establish baseline bleeding and treatment rates in a population of participants with VWD and act as comparator data for future clinical study outcomes.

开始日期2024-08-30

申办/合作机构

Hemab ApS

[+1]

NCT05916469 / Not yet recruitingN/AIIT

Heavy Menstrual Bleeding Progestin Treatment in Bleeding Disorders Study

The goal of this multicenter prospective observational study and registry of U.S. adolescents and young adults with heritable bleeding disorders is to determine the bleeding outcomes, satisfaction, hemostatic parameter changes, and patient reported quality of life after 6 months of use of either of two commonly used hormonal treatments for menstrual suppression - levonorgestrel intrauterine device (LNG-IUD) and norethindrone acetate (NETA). Under this application we will compare the two treatments and compare outcomes after LNG-IUD treatment results to a control group without a bleeding disorder, with the goal of determining the benefits and expected outcomes of these treatment options for this population.

开始日期2024-07-01

申办/合作机构

Oregon Health & Science University

[+1]

100 项与冯·维勒布兰德病相关的临床结果

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100 项与冯·维勒布兰德病相关的转化医学

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0 项与冯·维勒布兰德病相关的专利（医药）

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6,333

项与冯·维勒布兰德病相关的文献（医药）

2024-12-31·Renal Failure

Rare renal proximal tubular dysfunctions in primary biliary cholangitis

Article

作者: Ye, Wenling ; Qin, Yan ; Wen, Yubing ; Chen, Limeng ; Ye, Wei ; Li, Xuemei ; Zhang, Fengchun ; Zheng, Ke ; Guo, Tianchen ; Shi, Xiaoxiao

INTRODUCTIONRenal involvement of primary biliary cholangitis (PBC) usually presents as distal renal tubular acidosis. Proximal tubular (PT) dysfunctions in PBC were rarely reported with unclear clinicopathological characteristics and renal prognosis.METHODSWe identified 11 cases of PBC with PT dysfunctions (PBC-PT). Their medical document, kidney pathology, and follow-up data were retrospectively reviewed and analyzed.RESULTSThe 11 PBC-PT patients were mainly middle-aged (57.8 ± 5.2 years) females (81.8%). Most of them were asymptomatic PBC (7, 63.6%) with a high prevalence of elevated serum immunoglobulin M (IgM, 81.8%) and G (IgG, 54.5%) levels. In the kidney, they had a mean estimated glomerular filtration rate (eGFR) level of 46.54 ± 23.03 ml/min/1.73m², and 81.8% of them had eGFR below 60 ml/min/1.73m². They showed different degrees of PT dysfunctions, including hyperuricosuria, hypouricemia, normoglycemic glycosuria, generalized aminoaciduria, hyperphosphaturia, and hypophosphatemia. Their kidney pathology showed tubulointerstitial nephritis with lymphoplasmacytic infiltrates, brush border defects, and proximal tubulitis. After glucocorticoids treatment, the PT dysfunctions manifesting as hypophosphatemia, hypouricemia, and renal glycosuria all recovered, and the eGFR levels were improved from 43.24 ± 19.60 ml/min/1.73m² to 55.02 ± 21.14 ml/min/1.73m² (p = 0.028), accompanied by significant improvements of serum IgM levels (from 5.97 ± 4.55 g/L to 2.09 ± 1.48 g/L, p = 0.019).CONCLUSIONSThe PT dysfunctions were rare in PBC patients, and glucocorticoids treatment could benefit the improvements of eGFR and tubular functions.

2024-11-01·Journal of Thrombosis and Haemostasis

Unravelling the spectrum of von Willebrand factor variants in quantitative von Willebrand disease: results from a German cohort study

Article

作者: Yadegari, Hamideh ; Scholz, Ute ; Halimeh, Susan ; Pötzsch, Bernd ; Pezeshkpoor, Behnaz ; Knöfler, Ralf ; Liebscher, Karin ; Müller, Jens ; Olivieri, Martin ; Richter, Heinrich ; Trautmann-Grill, Karolin ; Pavlova, Anna ; Krahforst, Alexander ; Oldenburg, Johannes ; Trobisch, Heiner

BACKGROUNDVon Willebrand disease (VWD), the most prevalent hereditary bleeding disorder, results from deficiency of von Willebrand factor (VWF).OBJECTIVESThis large cohort study aims to offer a comprehensive exploration of mutation spectra and laboratory features in quantitative VWF deficiencies, shedding light on genetic underpinnings and genotype-phenotype associations.METHODSOur cohort consisted of 221 Caucasian index patients with quantitative VWD, along with 47 individuals whose plasma VWF levels fell within the lower normal boundaries (50-70 IU/dL). We conducted comprehensive VWF assays and genetic analyses, encompassing VWF gene sequencing, copy number variation investigations, and bioinformatic assessments.RESULTSFollowing International Society on Thrombosis and Haemostasis-Scientific and Standardization Committee VWF guidelines, 77 index patients were characterized as having type 1 VWD (VWF antigen [VWF:Ag] < 30 IU/dL), 111 as having type 1 VWD (VWF:Ag, 30-50 IU/dL), and 33 as having type 3 VWD. Mutation detection rates were 88%, 65%, and 92%, respectively. Notably, blood group O overrepresentation was evident in type 1 with VWF:Ag of 30 to 50 IU/dL, particularly among mutation-negative patients, suggesting a potential causal role of blood group O. A total of 223 VWF variants, comprising 147 distinct variations, were identified in quantitative VWD patients, of which 57 were novel variants (39%). Additionally, approximately 70% of individuals with VWF levels within the lower normal boundaries (50-70 IU/dL) displayed VWF variants.CONCLUSIONOur data advance our understanding of the molecular mechanisms underlying quantitative VWD, offering valuable insights for future research and clinical management. Distinct mutation patterns were observed among subgroups, particularly the contrast between type 1 VWD (VWF:Ag < 30 IU/dL) and type 1 VWD (VWF:Ag, 30-50 IU/dL), an area with limited prior investigation.

2024-11-01·International Journal of Oral and Maxillofacial Surgery

Matched cohort study evaluating the hemostatic efficacy of fibrin sealant versus conventional approaches following dental surgery in patients with hemophilia

Article

作者: Pai, N ; Gujar, H ; Dhaimade, P ; Chaudhari, V L ; Shanmukaiah, C ; Raj, J P

Patients with hemophilia frequently require supplementary interventions, either invasive (suturing, gel foam, or cauterization) or non-invasive (fibrin sealant/glue), to attain hemostasis post dental procedures. This study aimed to compare the efficacy of fibrin sealant against traditional methods for achieving hemostasis post dental surgery. The medical records of patients with factor VIII or IX deficiency, or von Willebrand disease, who underwent dental procedures in the Department of Dentistry, Seth GSMC and KEM Hospital, were evaluated for inclusion in this retrospective matched cohort study. Cohort-1 included those treated with a fibrin sealant (Tisseel Lyo) with/without traditional hemostatic measures post-procedure, while cohort-2 (controls) included those in whom no fibrin sealant was used. A total of 128 patients, 64 in each group, were evaluated. There was no statistically significant difference in demographics, disease-related variables, dental complaints, or preoperative treatment given between the groups. However, there was a significant reduction (P < 0.001) in the requirement for secondary procedures for hemostasis (suturing, gel foam application, and/or cauterization) and postoperative requirement for factor replacement (P = 0.003) in the fibrin glue group as compared to the controls. In this study, fibrin sealant demonstrated superior efficacy in mitigating the necessity for active hemostasis control.

106

项与冯·维勒布兰德病相关的新闻（医药）

2024-09-27

·美通社

融合本土智慧共创医疗未来：武田中国创新挑战赛概念性验证路演圆满落幕

上海 2024年9月27日 /美通社/ -- 近期，作为武田中国创新孵化器TakedaSpark成果展示的一环，第三届武田中国创新挑战赛概念性验证(PoC)结果路演在上海成功举办，五支精英团队在此次路演中展示了各自在数字医疗领域的前沿创新方案，旨在通过专业评估和验证，探索方案的商业潜力和实际应用价值，为商业化落地和患者受益打下坚实基础。武田制药全球高级副总裁、武田中国总裁单国洪，武田中国数字化科技团队负责人顾青，普华永道中国并购咨询服务合伙人费颖，埃森哲大中华区战略与咨询董事总经理万琳，亚马逊云科技医疗及生命科学行业方案高级总监黄庆春，上海生物医药创新转化基金投资人陈宇城及其他合作伙伴代表出席此次活动，并给予参赛队伍指导和建议。今年，武田迎来在华发展30周年。深耕中国30年来，武田中国不仅完成了新药研发、生产、供应链管理到商业化运营的完整药品价值链，还持续拓展本土朋友圈，加强与中国本土伙伴的创新合作，推动中国生物医药与数字化大健康产业高质量发展，与中国国家战略同频共振，助力医药行业加速"发展新质生产力"。武田中国创新挑战赛以及路演活动的举办是在武田"拓维中国"战略指导下，深耕中国市场的关键举措；同时，也进一步彰显武田对中国市场的坚定信心和不渝承诺。立足患者需求，持续探索数字化创新方案本次路演活动经过六个月的精心筹备，武田中国业务部门与初赛入围的本土创新科技企业共同组成的团队携手深入研究和实践，针对武田中国所专注的关键治疗领域，将数字化技术与疾病的全程管理相结合，不仅涵盖疾病的早期筛查服务，也深入到长期监测的各个场景。在参与路演的项目中，"多模态lgs脑电智能筛查平台"和"UC智能动态早诊系统"聚焦疾病早筛诊断领域，旨在提升医生的诊治效率、降低漏诊误诊发生频率；"Companion Care-癫痫智能数字化管理平台"则切实地看到了癫痫患者的痛点，在慢病管理上展现出了卓越的创新思维和优秀的实践能力；"AI智能对话-偏差编写赋能系统"与"vWD(血管性血友病)智慧筛查诊断系统"分别展现了大数据与AI技术在制药生产和疾病诊断方面的应用潜力，彰显了"时代科技"在医药行业的巨大潜力。武田中国数字化科技团队负责人顾青先生在路演活动上强调："数字化医疗不仅是国家政策的导向，更是未来发展的必然趋势。"他指出，在过去数年中，武田中国在多个领域和应用场景中均取得了突破性进展。第三届武田中国创新挑战赛精心设计，旨在满足武田中国在市场拓展、患者服务、医学事务、新产品研发以及药品生产等关键领域的数字化需求。这一赛事全面覆盖了武田中国在中国的整体价值链，凸显了数字化科技与各业务领域之间的紧密联系和协同效应。顾青表示，他相信通过这些优秀的创新项目，武田中国能够实现更高效的成果转化，最终造福广大患者。武田中国数字化科技团队负责人顾青致辞共创未来：数字化医疗领域的战略联盟与创新生态第三届武田中国创新挑战赛以及路演活动的圆满举办，不仅是对参赛队伍创新成果的检阅和肯定，更标志着武田中国在推动医疗行业数字化转型和与本土创新生态圈共创数字健康新未来的征程上再次迈出了坚实的步伐，为医疗行业的持续进步和患者福祉的提高奠定了坚实的基础。武田中国创新挑战赛概念性验证路演活动现场 TakedaSpark创始于2021年，是武田中国旗下开放式的创新孵化平台，致力于通过本土创新生态圈孵化共创的模式，探索高附加值的创新医疗方案和规模化的落地场景，为患者和医疗工作者以及医院提供优质的医疗创新服务。自首届赛事以来，共吸引200家本土创新企业报名，先后有18个创新方案进入最终的概念性验证(PoC)阶段 [1] 。在往届成功的基础上，第四届武田中国创新孵化器(TakedaSpark)于今年5月再次出发，并按计划进入筛选和评估阶段，挑选出最具潜力和价值的创新项目。新一届TakedaSpark 将继续聚焦数字化医疗，致力于构建一个行业领先的创新开放合作平台，以"合作、共赢、落地"作为核心战略，与全球顶尖创新团队紧密协作，共同探索医疗数字化的广阔天地，提供更高质量的治疗方案，以创新的力量照亮患者的未来，为他们带来更加健康和光明的生活前景。随着中国医疗科技领域的飞速发展，数字化技术已成为引领医药行业转型和创新的核心力量。作为数字医疗领域的先锋和积极倡导者，在全球战略指引和宏观市场背景下，武田中国始终致力于深化与中国本土生态系统的创新协作，更加开放、主动地拥抱数字化变革，加速数字化转型的步伐，与各方合作伙伴共同构建一个充满活力的创新生态圈，以推动行业进步并造福患者。审批编号：C-ANPROM/CN/CORP/0472 审批日期：2024年9月声明 1. 本资料仅为介绍企业业务及经营活动等情况，并不以宣传任何公司产品和/或服务为目的，更不应被理解为就任何药物、医疗器械及治疗方案的选择提供任何意见或建议。 2. 如欲了解任何公司产品、疾病和/或诊疗等相关信息，请务必咨询医疗卫生专业人士。注释说明 [1] ．数据来源：源自武田内部数据统计关于武田制药武田制药以"为人类创造健康生活，为世界缔造美好未来"为使命。我们专注于消化和炎症性疾病、罕见病、血液制品、肿瘤、神经科学及疫苗等关键治疗领域，着力研发并为患者带来突破性的创新疗法。我们的目标是携手合作伙伴，通过我们动态且多样化的产品管线，不断改善患者体验，并拓展对前沿性治疗方案的探索。武田制药总部位于日本，作为一家以价值观为基础、以研发为驱动的全球化生物制药公司，我们始终致力于兑现对患者、员工和地球的承诺。我们遍布全球约80多个国家和地区的员工肩负着相同的使命，始终践行着两个多世纪以来形成的价值观。更多信息，请访问https:\/\/www.takeda.com. 关于武田中国武田制药于1994年进入中国，武田中国总部位于上海，在中国的主要业务中心位于北京、上海、天津、广州等城市及香港特别行政区，并在全国各主要城市设有办事处，目前在中国拥有约2,800名员工。随着中国经济的发展和对医疗保健需求的不断增长，中国已经成为武田全球重要的战略市场之一。 2020年，武田中国启动"武聚未来"五年战略计划，持续加码中国市场，致力于满足中国市场未尽的医疗需求。至2025年，武田中国将在中国上市超过15款创新药，惠及超过1,000万中国患者。2021年9月，武田中国地区总部入驻上海浦东前滩，揭开了在华发展的又一新篇章。2022年，武田制药宣布启动"拓维中国"战略，充分释放中国这一全球第二大医药市场的全部潜力。至2030年，中国将成为武田全球第二大市场。前瞻性声明本稿件及与之相关的所分发的任何资料可能含有与武田未来业务、未来状况和运营业绩有关的前瞻性陈述、看法或意见，包括武田的预估、预测、目标和计划。前瞻性陈述常常包含但不限于下列措辞，例如"目标"、"计划"、"认为"、"希望"、"继续"、"预计"、"旨在"、"打算"、"确保"、"将"、"可能"、"应"、"会"、"或许"、"预期"、"估计"、"预测"或类似表述或其否定形式。本文中的前瞻性陈述仅基于武田截至发布日期的估计和假设。此类前瞻性声明并非是武田或其管理层对未来业绩所做的任何保证，并涉及已知和未知的风险、不确定性和其他因素，包括但不限于：武田全球业务所面临的经济形势，包括日本和美国的宏观经济环境；竞争压力和发展情况；适用法律法规的变动；产品开发项目的成功或失败；监管当局的决策或做出决策的时机；利率和汇率波动；有关已售产品或候选产品安全或功效的索赔或疑问；已收购公司的合并后整合努力的时机和影响；以及能否剥离对武田运营非核心的资产和任何此类剥离的时机，所有这些都可能会导致武田的实际业绩、表现、成就或财务状况与此类前瞻性陈述所描述或暗指的任何未来业绩、表现、成就或财务状况发生重大偏差。关于以上及可能影响武田结果、业绩、成就或财务状况的其他因素的更多信息，请参阅武田向美国证券交易委员会提交的Form 20-F最新年报"第3项.关键信息—D.风险因素"和其他报告，具体请查阅武田网站https:\/\/www.takeda.com\/investors\/reports\/sec-filings\/或www.sec.gov。武田的未来结果、业绩成就或财务地位可能与前瞻性陈述所言传或意会的内容有实质性差距。收到这一新闻稿的个人不应对任何前瞻性陈述寄予不适切的依赖。武田没有义务更新本新闻稿中的任何前瞻性陈述或公司可能发布的任何其他前瞻性陈述，除非是法律或证券交易规则所要求。历史业绩并不能代表未来业绩，而且本新闻稿中的武田业绩并不能指代，也并非是武田未来业绩的预估、预测或推测。

并购

2024-09-08

·生物探索

Immunity | 抗体和补体是血栓形成的关键驱动因素

引言抗体是体液免疫防御的主要组成部分，用以清除衰老细胞，维持机体稳态【1】。除此之外，抗体还可以用于治疗恶性和炎症性疾病【2】。一些临床证据表明，抗体，无论其来源 (内源性/外源性) 或特异性 (多克隆/抗原特异性) 如何，都可促使血栓形成。抗体治疗，包括且不限于抗胸腺细胞球蛋白、抗表皮生长因子受体 (EGFR)，抗血管内皮生长因子受体 (EGFR) 以及静脉注射免疫球蛋白 (IVIG)【3】，都与高凝状态和血栓并发症有关。这些发现是否与抗体的特定作用模式有关 (也就是它们的抗原特异性)，或者抗体本身是否具有血栓性尚不清楚【4-7】。不仅是输入人体的外源性治疗性抗体，而且内源性抗体的产生失调也与血栓形成风险增加有关，比如最近报道了多例疫苗诱导的免疫性血栓性血小板减少症、肝素诱导的血小板减少症、抗磷脂综合征、多发性骨髓瘤，以及目前病因不明的单克隆免疫球蛋白病。尽管上述病例还没有找到共性，但是这些内源性抗体的血栓前作用似乎与靶抗原有关，且潜在机制仍不清楚。与抗体类似，补体系统 (抗体的主要效应臂) 也与血栓形成有关:正常人群补体C3浓度升高与静脉血栓栓塞的风险增加有关。在包括COVID-19在内的传染病中，补体也与血栓形成事件有关。此外，补体活化与血栓性血小板减少性紫癜的血栓前状态有关。在系统性红斑狼疮或原发性自身免疫性溶血性贫血患者中，广泛的自身抗体驱动的补体激活与深静脉血栓形成和补体因子在血小板上沉积的高风险相关。除了抗体触发的补体激活外，抗体非依赖性补体激活也与血栓形成前状态相关：突发性夜间血红蛋白尿 (CD55和CD59) 或非典型溶血性尿毒症综合征 (如因子H) 中补体调节蛋白的基因突变也与血栓形成有关。靶向补体药物在治疗上述这些疾病的同时，也能降低血栓形成的风险。但是，抗体和补体驱动血栓形成的关键机制仍没有研究透彻。 2024年9月2日，来自德国慕尼黑大学的Konstantin Stark等人在Immunity上发表了文章Antibodies and complement are key drivers of thrombosis，发现抗体和补体是小鼠静脉血栓形成的关键驱动因素，治疗性补体抑制可以防止血栓形成，而不会对止血产生负面影响。静脉血栓栓塞 (VTE) 是一种常见的致命疾病，尽管采取了预防措施，但发病率仍在增加。临床观察表明抗体浓度升高或基于抗体的治疗与血栓事件有关。然而，抗体如何促成血栓形成尚不清楚。在这里，作者揭示了抗体和补体如何共同作为体内血栓形成的中枢介质。作者发现，抗体的作用独立于它们的抗原结合功能，并且发生在两个阶段，每个阶段都由不同种类的抗体主导。首先是第一阶段，深静脉血栓形成期间的血流减少，允许IgM结合内皮细胞，从而形成Toso和聚合免疫球蛋白受体介导的内皮细胞活化，并启动内皮细胞暴露血管性血友病因子和选择素P所诱导的血小板和白细胞募集。其次是第二阶段，血小板活化之后，可以提供促凝剂表面，促进纤维蛋白依赖性和硫酸软骨素A依赖性的IgG沉积，且这一阶段无需考虑抗体的抗原特异性以及Fc受体参与与否。上述两个阶段启动了补体驱动的血小板激活和髓系白细胞募集的恶性循环，最终导致血栓形成。另外，作者还确定，静脉停滞和covid -19免疫血栓失调的人类中存在上述机制。并且，这种抗体驱动的血栓形成可以通过药物靶向补体来预防。综上所述，作者的研究结果揭示了一种抗体驱动血栓形成途径，该途径通过IgM受体和聚合免疫球蛋白受体介导的内皮细胞活化，以及随后的IgG介导的补体激活启动血栓形成恶性循环。作者的工作表明，靶向这一血栓形成前轴为血栓形成提供了一种创新的预防和治疗选择，重要的是，它规避了与传统抗凝治疗相关的出血风险，为靶向血栓形成而不影响止血开辟了创新途径。参考文献 1. Ehrenstein, M.R., and Notley, C.A. (2010). The importance of natural IgM: scavenger, protector and regulator. Nat. Rev. Immunol. 10, 778–786. https://doi-org.libproxy1.nus.edu.sg/10.1038/nri2849. 2. Elvin, J.G., Couston, R.G., and van der Walle, C.F. (2013). Therapeutic antibodies: market considerations, disease targets and bioprocessing. Int. J. Pharm. 440, 83–98. https://doi-org.libproxy1.nus.edu.sg/10.1016/j.ijpharm.2011.12.039. 3. Aggarwal, R., Charles-Schoeman, C., Schessl, J., Bata-Cso ̈rgo}, Z., Dimachkie, M.M., Griger, Z., Moiseev, S., Oddis, C., Schiopu, E., Vencovsky ́, J., et al. (2022). Trial of Intravenous Immune Globulin in Dermatomyositis. N. Engl. J. Med. 387, 1264–1278. https://doi-org.libproxy1.nus.edu.sg/10. 1056/NEJMoa2117912. 4. Alahmari, A.K., Almalki, Z.S., Alahmari, A.K., and Guo, J.J. (2016). Thromboembolic Events Associated with Bevacizumab plus Chemotherapy for Patients with Colorectal Cancer: A Meta-Analysis of Randomized Controlled Trials. Am. Health Drug Benefits 9, 221–232. 5. Ammann, E.M., Jones, M.P., Link, B.K., Carnahan, R.M., Winiecki, S.K., Torner, J.C., McDowell, B.D., Fireman, B.H., and Chrischilles, E.A. (2016). Intravenous immune globulin and thromboembolic adverse events in patients with hematologic malignancy. Blood 127, 200–207. https://doi-org.libproxy1.nus.edu.sg/10.1182/blood-2015-05-647552. 6. Cumpelik, A., Gerossier, E., Jin, J., Tsakiris, D., Dickenmann, M., Sadallah, S., Schifferli, J.A., and Zecher, D. (2015). Mechanism of Platelet Activation and Hypercoagulability by Antithymocyte Globulins (ATG). Am. J. Transplant. 15, 2588–2601. https://doi-org.libproxy1.nus.edu.sg/10.1111/ ajt.13316. 7. Daniel, G.W., Menis, M., Sridhar, G., Scott, D., Wallace, A.E., Ovanesov, M.V., Golding, B., Anderson, S.A., Epstein, J., Martin, D., et al. (2012). Immune globulins and thrombotic adverse events as recorded in a large administrative database in 2008 through 2010. Transfusion 52, 2113– 2121. https://doi-org.libproxy1.nus.edu.sg/10.1111/j.1537-2995.2012.03589.x. 8. Gupta, D., Shukla, P., Bisht, S.S., Bhatt, M.L., Pant, M.C., and Srivastava, K. (2011). Deep vein and artery thrombosis associated with cetuximab- based chemoradiotherapy. Indian J. Pharmacol. 43, 478–480. https:// doi.org/10.4103/0253-7613.83135. https://doi-org.libproxy1.nus.edu.sg/10.1016/j.immuni.2024.08.007 责编|探索君排版|探索君文章来源|“BioArt” End 往期精选围观一文读透细胞死亡(Cell Death) | 24年Cell重磅综述（长文收藏版）热文 Cell | 是什么决定了细胞的大小？热文 Nature | 2024年值得关注的七项技术热文 Nature | 自身免疫性疾病能被治愈吗？科学家们终于看到了希望热文 CRISPR技术进化史 | 24年Cell综述

疫苗

2024-09-05

·Business Wire

Pathway to Cures Announces Two New Board Members

NEW YORK--( BUSINESS WIRE )--Pathway to Cures (P2C) announces two new members to its board of directors, Sharon Richardson, PhD , a Director on the National Bleeding Disorders Foundation Board, and Philip M. Gattone, M.Ed. , President and CEO of the National Bleeding Disorders Foundation. Pathway to Cures is the venture philanthropy fund for the National Bleeding Disorders Foundation (NBDF) focused on investing in emerging biotech companies developing cures, therapies, or enabling technologies in support of the inheritable blood and bleeding disorders community. Inheritable blood and bleeding disorders include rare and ultra-rare blood disorders, hemophilia A and B, von Willebrand disease, Sickle Cell disease, anemia, clotting disorders, and other hematological disorders, affecting more than 20 million people worldwide. Sharon Richardson, PhD, is a result-driven strategist with 28 years of experience in the pharmaceutical industry as a medical affairs leader committed to creating healthier communities through scientific exchange, patient advocacy, and community engagement. “P2C offers a unique opportunity for the National Bleeding Disorders Foundation to leverage their 75 years of serving people living with blood and bleeding disorders,” said Sharon Richardson. “It is a privilege to lend my time and assistance to advance the science and treatment options available to our community and to work alongside this talented and dedicated group of staff and volunteers.” “Sharon has led a life of selfless service to others and has used her strong leadership skills to make a difference for our community.” said Phil Gattone “We are thrilled to have Sharon join the Pathway to Cures Board and look forward to working with her to make a positive impact in more lives.” Philip M. Gattone, M.Ed., joined the National Bleeding Disorders Foundation as president and chief executive officer earlier this year. Gattone is a seasoned leader dedicated to amplifying the impact of nonprofit organizations through his distinctive blend of leadership and strategic insight. He has served as a senior executive in local, regional, national, and global nonprofit organizations and healthcare companies. “We’re honored and delighted to have Sharon and Phil supporting our efforts for the inheritable blood and bleeding disorders community!” said Teri Willey, Managing Director of the Venture Philanthropy Fund “Their knowledge and experience will enhance our ability to ensure there are more therapies and cures available for our community.” About Pathway to Cures Pathway to Cures (P2C) is the venture philanthropy fund of the National Bleeding Disorders Foundation created to accelerate the development of cures across all inheritable blood and bleeding disorders. In collaboration with other organizations, P2C invests in innovative therapies and technologies, leveraging the deep resources and scientific community relationships of the National Bleeding Disorders Foundation. By reinvesting proceeds from investments back into the Fund, P2C will amplify the investment impact, support promising biotech companies, and build a portfolio of investments that further the mission of the National Bleeding Disorders Foundation. For more information, visit www.pathwaytocures.org or contact Renee Peck at rpeck@pathwaytocures.org . About National Bleeding Disorders Foundation The National Bleeding Disorders Foundation (NBDF) is dedicated to finding cures for inheritable blood and bleeding disorders and addressing and preventing these disorders' complications through research, education, and advocacy, enabling people and families to thrive. Today, NBDF serves people across the United States with all bleeding disorders, including hemophilia, von Willebrand disease, rare factor deficiencies, and platelet disorders. The foundation also supports a network of 50 chapters across the country. To learn more, visit www.bleeding.org and follow NBDF across social media @nbd_foundation.

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