This is a double-blind, crossover food challenge study using pork with and without α-gal in patients with a clinical diagnosis of gastrointestinal (GI)- α-gal allergy, and to investigate the pathophysiology underlying their symptoms.

开始日期2023-10-17

申办/合作机构

The University of North Carolina at Chapel Hill

[+1]

NCT05067868 / Active, not recruiting临床4期

A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Agalsidase Alfa (r-DNA Origin) (Replagal™) in Indian Children and Adults With Fabry Disease

The main aim of this study is to learn more about the safety profile of Replagal.
Participants will receive Replagal every 2 weeks at the clinic for about 1 year.

开始日期2022-11-01

申办/合作机构

Shire Plc

NCT04974749 / Completed临床3期

An Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of REPLAGAL® in Treatment-naïve Chinese Subjects With Fabry Disease

The main aim of the study is to assess the safety of REPLAGAL. Study participants will receive REPLAGAL as an intravenous infusion every other week for 52 weeks. Participants will visit their study clinic many times throughout the study.

开始日期2022-05-06

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

100 项与阿加糖酶α 相关的临床结果

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100 项与阿加糖酶α 相关的转化医学

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100 项与阿加糖酶α 相关的专利（医药）

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1,166

项与阿加糖酶α 相关的文献（医药）

Orphanet journal of rare diseases

Reducing agalsidase beta infusion time in Fabry patients: low incidence of antibody formation and infusion-associated reactions in an Italian multicenter study

Article

作者: Pennacchiotti, Fabio ; Pieruzzi, Federico ; Tartaglia, Luciano ; Cianci, Vittoria ; Battaglia, Yuri ; Lanzillo, Chiara ; Mignani, Renzo ; Americo, Claudio ; Sapuppo, Annamaria ; Marchi, Giacomo ; Aucella, Filippo

Abstract:

Background:

Fabry disease is a rare progressive X-linked lysosomal storage disease caused by mutations in the GLA gene that encodes α-galactosidase A. Agalsidase beta is a recombinant enzyme replacement therapy authorized in Europe at a standard dose of 1.0 mg/kg intravenously every other week at an initial infusion rate of ≤ 0.25 mg/min until patient tolerance is established, after which the infusion rate may be increased gradually. However, specific practical guidance regarding the progressive reduction in infusion time is lacking. This study investigated a new and specific protocol for reducing agalsidase beta infusion time in which a stable dosage of 15 mg/h is infused for the first four months, and the infusion rate is increased progressively from 15 to 35 mg/h for the subsequent four infusions. The shortest infusion time is reached after six months and maintained thereafter. The incidence of infusion-associated reactions (IARs) and the development of anti-drug antibodies were analyzed, and the disease burden and the clinical evolution of the disease at 12 months were evaluated.

Results:

Twenty-five of the 31 patients were naïve to enzyme or chaperone treatment at baseline and six patients had been switched from agalsidase alfa. The reduced infusion time protocol was well tolerated. Only one patient exhibited an IAR, with mild symptoms that resolved with low-dose steroids. Six patients globally seroconverted during treatment (4 with a classic phenotype and 2 with late-onset disease). All but three patients were seronegative at month 12. All patients were stable at the study’s end (FAbry STabilization indEX value < 20%); reducing infusion time did not negatively impact clinical outcomes in any patient. The perceived medical assessment showed that the quality of life of all patients improved.

Conclusions:

The study demonstrates that reducing agalsidase beta infusion time is possible and safe from both an immunogenic and clinical point of view. The use of a low infusion rate in the first months when the probability of onset of the development of antibodies is higher contributed to very limited seroconversion to antibody-positive status.

2024-01-24·Journal of the American Chemical Society

Linkage-Editing Pseudo-Glycans: A Reductive α-Fluorovinyl-C-Glycosylation Strategy to Create Glycan Analogs with Altered Biological Activities

Article

作者: Nagatake, Takahiro ; Saika, Azusa ; Nakahashi, Rintaro ; Kunisawa, Jun ; Moritsuka, Natsuho ; Kudo, Noyuri ; Usui, Kazuteru ; Moriyama, Takahiro ; Yoritate, Makoto ; Kusuhara, Wakana ; Takahashi, Shunji ; Chiba, Suzuka ; Ono, Shunsuke ; Tomooka, Katsuhiko ; Hirai, Go ; Tanabe, Riko ; Igawa, Kazunobu ; Kato, Naoki ; Matoba, Hiroaki ; Koshino, Hiroyuki ; Hidaka, Yu ; Kiya, Noriaki ; Ishikawa, Eri ; Yamasaki, Sho

The acetal (O-glycoside) bonds of glycans and glycoconjugates are chemically and biologically vulnerable, and therefore C-glycosides are of interest as more stable analogs. We hypothesized that, if the O-glycoside linkage plays a vital role in glycan function, the biological activities of C-glycoside analogs would vary depending on their substituents. Based on this idea, we adopted a "linkage-editing strategy" for the creation of glycan analogs (pseudo-glycans). We designed three types of pseudo-glycans with CH₂ and CHF linkages, which resemble the O-glycoside linkage in terms of bond lengths, angles, and bulkiness, and synthesized them efficiently by means of fluorovinyl C-glycosylation and selective hydrogenation reactions. Application of this strategy to isomaltose (IM), an inducer of amylase expression, and α-GalCer, which activates iNKT cells, resulted in the discovery of CH₂-IM, which shows increased amylase production ability, and CHF-α-GalCer, which shows activity opposite that of native α-GalCer, serving as an antagonist of iNKT cells.

2024-01-02·Angewandte Chemie (International ed. in English)

Diversification of a Novel α‐Galactosyl Ceramide Hotspot Boosts the Adjuvant Properties in Parenteral and Mucosal Vaccines

Article

作者: Davari, Mehdi D ; Vasco, Aldrin V ; Méndez, Yanira ; Wessjohann, Ludger A ; Guzmán, Carlos A ; Schulze, Kai ; Rivera, Daniel G ; Ebensen, Thomas ; Westermann, Bernhard ; Yousefi, Mohammad

Abstract:

The development of potent adjuvants is an important step for improving the performance of subunit vaccines. CD1d agonists, such as the prototypical α‐galactosyl ceramide (α‐GalCer), are of special interest due to their ability to activate iNKT cells and trigger rapid dendritic cell maturation and B‐cell activation. Herein, we introduce a novel derivatization hotspot at the α‐GalCer skeleton, namely the N‐substituent at the amide bond. The multicomponent diversification of this previously unexplored glycolipid chemotype space permitted the introduction of a variety of extra functionalities that can either potentiate the adjuvant properties or serve as handles for further conjugation to antigens toward the development of self‐adjuvanting vaccines. This strategy led to the discovery of compounds eliciting enhanced antigen‐specific T cell stimulation and a higher antibody response when delivered by either the parenteral or the mucosal route, as compared to a known potent CD1d agonist. Notably, various functionalized α‐GalCer analogues showed a more potent adjuvant effect after intranasal immunization than a PEGylated α‐GalCer analogue previously optimized for this purpose. Ultimately, this work could open multiple avenues of opportunity for the use of mucosal vaccines against microbial infections.

项与阿加糖酶α 相关的新闻（医药）

2024-06-20

·BioSpace

Prostate Drugs May Find New Use in Reducing Risk of Dementia: Study

Pictured: Concept art showing an elderly woman overlaid with an illustration of a brain/iStock, boonstudio Drugs commonly used for the treatment of enlarged prostate may also help lower the risk of dementia with Lewy bodies, according to a new observational study from the University of Iowa Health Care. Drawing from Merative MarketScan, one of the largest proprietary U.S. healthcare claims databases, University of Iowa researchers identified more than 643,000 men who were starting treatment for benign prostatic hyperplasia with one of six common therapies. Three of these drugs fell under the class of α-1 adrenergic receptor antagonists, better known as α-blockers, which work by inhibiting the contraction of smooth muscles. The researchers focused on terazosin, which is sold by Abbott under the brand name Hytrin, doxazosin, which is marketed by Pfizer as Cardura, and alfuzosin, which is branded as Uroxatral by Sanofi. Alpha-blocker drugs are known to have a unique side effect: they activate a key enzyme involved in the production of ATP, which is the molecular form of cellular energy. In the brain, these drugs can help boost the production of energy and fight neurodegeneration, the University of Iowa scientists hypothesized. As active comparators, the researchers also followed men in Merative MarketScan who were being treated with tamsulosin or with the 5α-reductase inhibitors (5ARI) finasteride and dutasteride, all of which are not known to affect energy production in the brain. The men included in the study had no history of dementia with Lewy bodies (DLB). Participants were then followed until they exited the database or until they developed DLB. After an average follow-up of around three years, the study found that men who were initiated on the three alpha-blocker drugs were 40% less likely to develop DLB than comparators who were on tamsulosin. Compared with men whose enlarged prostates were being treated with 5ARI agents, the risk of DLBA was 27% lower in those taking alpha-blocker drugs. Both effects were statistically significant. By contrast, DLB had a statistically similar likelihood of developing in men being managed with tamsulosin and 5ARIs. These results were robust to several sensitivity analyses, according to researchers. The University of Iowa scientists published their findings Wednesday in Neurology, the medical journal of the American Academy of Neurology. “If terazosin and these similar medications can help slow this progression—if not outright preventing the disease—this would be important to preserving cognitive function and quality of life in people with DLB,” Jacob Simmering, assistant professor of internal medicine at the University of Iowa and lead study author, said in a statement. However, the researchers caution that the study is simply observational in design and only shows that there is a statistical correlation between the use of alpha-blocker drugs and the reduced risk of DLB. It does not establish causation nor does it show any directionality of association, they said. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

临床结果

2024-04-24

·FiercePharma

Chiesi survey shows how Fabry drugs are failing patients, providing road map for ousting incumbents

Survey results speak to why Chiesi sees a need for Elfabrio. A Chiesi survey has found widespread unmet needs among Fabry disease patients taking drugs such as Sanofi’s Fabrazyme, pointing to how the Italian drugmaker can muscle in on the market. Chiesi won FDA approval for the Fabry disease drug Elfabrio last year. Patients already had access to Sanofi’s old stager Fabrazyme and Amicus Therapeutics' more recently approved oral challenger Galafold. But Chiesi identified a persistent unmet need, leading it to pay Protalix BioTherapeutics $50 million upfront plus $45 million to cover development costs to secure the rights to a Fabry prospect. Seeking to understand the unmet need, Chiesi and its collaborators surveyed 280 adults in the U.S. and Canada about their experience of living with Fabry. The researchers recently published the findings in the Orphanet Journal of Rare Diseases. Most, 84%, of the respondents were taking an enzyme replacement therapy (ERT) such as Fabrazyme or Takeda’s Replagal. Eleven percent of respondents were taking chaperone therapy, namely Galafold. Most of the people had unmet needs despite being on one of the approved treatments. The most common symptoms were low energy, tingling and pain in the hands or feet. Between 60% and 72% of respondents had those symptoms. More than half of the Fabry patients had ringing in their ears or hearing loss, general body pains or stomach pains. For 51% of respondents, the symptoms were hard to control or bothersome. Symptoms temporarily worsened between infusions in around half of respondents. However, only 48% of those patients reported the worsening to their physicians. In most cases, the doctor neither changed the patient’s treatment nor prescribed them a medicine to manage the symptoms. The survey results speak to why Chiesi sees a need for Elfabrio. Chiesi’s ERT performed similarly to Fabrazyme on the primary endpoint in a head-to-head phase 3 clinical trial. Elfabrio has a longer half-life, though, suggesting symptoms may be less likely to worsen between infusions, and its PEGylated design and other elements could improve tolerability and lower the rate of infusion-related reactions. Chiesi posted sales of 3 billion euros ($3.2 billion) in 2023, supported by 65% growth at the rare disease unit that sells Elfabrio. The statement lacks details of Elfabrio sales. Protalix generated $17.5 million from sales of Elfabrio to Chiesi last year.

临床3期临床结果上市批准

2023-12-12

·今日头条

蜱虫叮咬使人对红肉过敏？JAMA解析α-gal综合征

12月12日的《热心肠日报》，我们解读了 10 篇文献，关注：α-gal综合征，食物过敏，肠道病原体，AHR配体，腹泻，ILC3，关节炎，肺纤维化。 JAMA：一文读懂α-gal综合征 JAMA——[120.7] ① 蜱虫叮咬时被咬个体会被注入半乳糖-α-1,3-半乳糖（α-gal）形成抗体，α-gal存在于大多数哺乳动物中，被咬个体食用这些动物产品时，可能会发生过敏反应，被称为α-gal综合征；② 过敏症状在摄入动物产品后2至6小时发作，可能很轻微，也可危及生命；③ 当个体对红肉等动物产品产生迟发性过敏反应并且血液检测出α-gal抗体时则可被确诊；④ 轻度症状用抗组胺药物治疗，严重时通过肌肉注射肾上腺素来治疗；⑤ 避免蜱虫叮咬即可预防α-gal综合征。【主编评语】 α-gal是一种双糖，广泛存在于猪、牛、羊等动物的肌肉和内脏中，蜱虫叮咬后能引起α-gal特异性IgE水平升高，再当摄入红肉或内脏引起IgE介导的过敏反应，称之为α-gal综合征。JAMA最新文章就α-gal综合征的症状、诊断、治疗、预防等方面进行简要归纳。（@RZN）【原文信息】 What Is Alpha-Gal Syndrome? 2023-12-08, doi: 10.1001/jama.2023.23097 Cell子刊：膳食补充AHR配体或可抵御隐孢子虫感染 Cell Host and Microbe——[30.3] ① 隐孢子虫感染回肠，引起炎症、上皮细胞损伤并影响上皮细胞分化，但不影响结肠；② 造血细胞特异性芳香烃受体（AHR）缺陷小鼠易受隐孢子虫感染；③ 上皮AHR信号传导对于控制寄生虫生长来说是可有可无的；④ CD8+上皮内淋巴细胞（IELs）中AHR表达是其维持和细胞毒性所必需的，这些CD8+IELs可以有效控制宿主体内隐孢子虫的生长；⑤ 新生小鼠进行膳食补充AHR配体吲哚-3-甲醇可增加CD8+IELs，可保护小鼠免受隐孢子虫感染。【主编评语】隐孢子虫感染是儿童腹泻致死的主要原因，特别是在资源匮乏的环境中，隐孢子虫还会针对免疫功能低下、长期感染艾滋病毒和原发性免疫缺陷的人，目前没有疫苗或有效的治疗方法。Cell Host and Microbe最新研究性文章表明肠道CD8阳性上皮内淋巴细胞对隐孢子虫感染具有抵抗力，并且这一过程依赖于芳香烃受体AHR，并且补充AHR膳食配体可增强宿主对隐孢子虫病的免疫反应，从而预防疾病。（@RZN）【原文信息】 Dietary environmental factors shape the immune defense against Cryptosporidium infection 2023-12-04, doi: 10.1016/j.chom.2023.11.008 Nature子刊：医院获得性腹泻患者生物标志物有何特征？ Nature Communications——[16.6] ① 纳入169例医院获得性腹泻（HAD）患者，其中33例为艰难梭菌感染（CDI），通过表征肠群和代谢组学特征以确定生态失调标志物；② HAD、抗生素相关腹泻（AAD）和CDI患者与不同肠群和代谢物组成相关，延长抗生素暴露和联合抗生素治疗与以肠球菌科为主的菌群有关；③ 酪胺/酪氨酸比值升高和脯氨酸Stickland发酵可作为低多样性肠球菌显性AAD以及伴随肠球菌增殖和CDI生物标志物；④ 氨基酸脯氨酸和酪氨酸是CDI和万古霉素耐药肠球菌增殖的重要底物。【主编评语】医院获得性腹泻（HAD）很常见，通常与抗生素给药后肠道菌群和代谢组失调有关。艰难梭菌是最重要的抗生素相关性腹泻 (AAD) 病原体，但人们对AAD和艰难梭菌感染 (CDI) 与对比抗生素治疗相关的微生物群和代谢组知之甚少。近日，莫纳什大学研究人员在Nature Communications发表最新研究，揭示了医院获得性腹泻患者的生物标志物，发现酪胺/酪氨酸比值升高和脯氨酸Stickland发酵分别可作为低多样性肠球菌显性AAD以及伴随肠球菌增殖和CDI的生物标志物，值得关注。（@九卿臣）【原文信息】 Multi-omics analysis of hospital-acquired diarrhoeal patients reveals biomarkers of enterococcal proliferation and Clostridioides difficile infection 2023-11-25, doi: 10.1038/s41467-023-43671-8 感染致泻性大肠杆菌却无腹泻症状，原因何在？ Gut Microbes——[12.2] ① 利用厄瓜多尔腹泻病例对照研究中致泻性大肠杆菌（DEC）基因组和粪便宏基因组数据，研究与DEC腹泻症状相关的因素；② 有症状DEC感染者肠道中大肠杆菌相对丰度、毒力基因的数量和相对丰度均高于无症状DEC感染者；③ 无论是否腹泻或携带DEC，肠道菌群多样性相似且变形菌富集，而未感染者中拟杆菌和双歧杆菌富集；④ 有/无症状感染者的DEC分离株在毒力或辅助基因含量上无显著差异，且无与腹泻症状相关的系统发育信号。【主编评语】很多肠道感染如致泻性大肠杆菌（DEC）引起的感染没有腹泻症状，造成有/无腹泻症状的因素目前仍不清楚。近日，发表在Gut Microbes上的这篇文章，发现区分有/无症状DEC感染的信号：即大肠杆菌丰度、肠道菌群毒力基因含量及其分类单元具有明显差异。（@圆圈儿）【原文信息】 Why are so many enteric pathogen infections asymptomatic? Pathogen and gut microbiome characteristics associated with diarrhea symptoms and carriage of diarrheagenic E. coli in northern Ecuador 2023-11-22, doi: 10.1080/19490976.2023.2281010 Gasdermin D可为小鼠急性沙门氏菌肠道感染提供保护 PNAS——[11.1] ① 小鼠急性沙门氏菌（S.Tm）肠道感染中，GSDMA、C和E似乎是可有可无的，但GSDMD限制肠道组织和系统器官中的S.Tm负荷，控制肠道炎症动力学，以及在感染72小时前防止上皮破裂；② 只有骨髓来源的固有层细胞和肠上皮细胞（IEC）都表达GSDMD，这种保护才是完全的；③ 在没有GSDMD的情况下，受感染的IEC挤出也能进行，但GSDMD控制挤出IEC的渗透性和形态，影响挤出动力学，并促进整体粘膜屏障能力。【主编评语】 Gasdermins是参与细胞焦亡的重要蛋白，参与癌症和传染病（如急性鼠伤寒沙门氏菌（S.Tm）肠道感染）相关的各种免疫反应。PNAS近期发表文章，发现GSDMD是Gasdermins家族中唯一一个对小鼠急性沙门氏菌肠道感染提供保护，对粘膜组织防御常见肠道病原体具有多重作用。（@章台柳）【原文信息】 Gasdermin D is the only Gasdermin that provides protection against acute Salmonella gut infection in mice 2023-11-21, doi: 10.1073/pnas.2315503120 Cell子刊：肠道ILC3如何控制致病菌引发的肠炎？ Cell Reports——[8.8] ① 小鼠RORγt +细胞中HIF-1α对于防御柠檬酸杆菌诱导的结肠炎至关重要；② 先天性RORγt+ 细胞中HIF-1α缺乏会损害小鼠感染期间IL-22的诱导，从而加剧结肠炎；③ 感染柠檬酸杆菌后，小鼠HIF-1α促进ILC3中的IL-22，但不会促进其他先天RORγt+ 细胞中的IL-22；④ 柠檬酸杆菌感染导致小鼠结肠中TLR2诱导的IL-18产生，其转录上调ILC3中的HIF-1α，从而促进IL-22表达。【主编评语】 3型先天淋巴细胞ILC3对防御组织屏障免受病原体入侵至关重要，缺氧通过激活HIF影响肠道ILC3衍生细胞因子的产生，然而目前对体内感染过程中ILC3和其他先天RORγt+ 细胞中HIF-1α的控制机制知之甚少。近日，发表在Cell Reports上的这篇文章，发现ILC3中IL-18诱导的HIF-1α改善啮柠檬酸杆菌诱导的结肠炎。（@圆圈儿）【原文信息】 IL-18-induced HIF-1α in ILC3s ameliorates the inflammation of C. rodentium-induced colitis 2023-11-28, doi: 10.1016/j.celrep.2023.113508 肠道菌群保护“超级愈合者”小鼠抵抗创伤后骨关节炎 Annals of the Rheumatic Diseases——[27.4] ① 破坏半月板手术（DMM）可诱导创伤后骨关节炎（OA），在DMM之前将MRL小鼠的菌群移植到B6小鼠，将DMM后8周的OA组织病理学、滑膜炎和骨赘评分降低；② DMM后48h将MRL的菌群移植到B6，可改善OA结果，但DMM后1-2周进行移植则效果不佳；③ MRL的菌群移植到无菌小鼠（F1）并繁殖一代（F2），DMM诱导产生OA在F1和F2降低60%和30%；④ 几种盲肠微生物组分支与较好或较差OA结果相关；⑤ MRL移植到B6和MRL小鼠中，双阴性T细胞减少和CD25+CD4+T细胞增加。【主编评语】 Murphy-Roths Large（MRL）/MpJ“超级愈合者”小鼠品系可以保护其免受创伤后骨关节炎（OA）的影响。Annals of the Rheumatic Diseases近期发表的文章，发现肠道微生物组在一定程度上接到了MRL小鼠对OA的抵抗，并可通过菌群移植进行转移。移植所诱导的静息状态下免疫表型变化与OA保护相关。（@章台柳）【原文信息】 OA susceptibility in mice is partially mediated by the gut microbiome, is transferrable via microbiome transplantation and is associated with immunophenotype changes 2023-11-18, doi: 10.1136/ard-2023-224907 国内团队：肠菌代谢物与症状性手骨关节炎间有何关联？ EBioMedicine——[11.1] ① 通过两个独立队列探究肠菌代谢物与症状性手骨关节炎（SHOA）间的关联，在发现队列（n=1359）中，SHOA患者肠菌相关的色氨酸代谢功能改变；② SHOA患者血浆中5-羟基吲哚乙酸和5-羟色氨酸水平较高，而吲哚-3-乳酸（ILA）、3-羟基邻氨基苯甲酸和甲基吲哚水平较低；③ SHOA患者血浆ILA水平较低与肠菌色氨酸代谢功能相关，且色氨酸代谢功能与Bacteroides A mediterraneensis进一步相关；④ 通过验证队列（n=142）证实，较低水平的ILA与SHOA相关。【主编评语】骨关节炎是常见关节病，会引起患者的关节软骨受到损害，甚至引起患者的整个关节受累、蜕变和纤维化溃疡等。由于肠道菌群失调可通过影响宿主代谢引起炎症性疾病。近日，中南大学湘雅医院雷光华、曾超及团队在EBioMedicine发表最新研究，通过两个独立队列探究肠菌代谢物与症状性手骨关节炎（SHOA）间的关联，发现色氨酸生物合成和色氨酸代谢物微生物功能的改变，特别是吲哚-3-乳酸水平的降低，与SHOA相关，值得关注。（@九卿臣）【原文信息】 Association between gut microbiome-related metabolites and symptomatic hand osteoarthritis in two independent cohorts 2023-11-24, doi: 10.1016/j.ebiom.2023.104892 长期摄入食品添加剂TiO2或促食物过敏 Allergy: European Journal of Allergy and Clinical Immunology——[12.4] ① 母鼠从受孕前到断奶期间喂食对照饮食或富含食品级二氧化钛（fg-TiO2）饮食，且其后代在断奶后接受相同饮食，通过评估肠道屏障成熟度和微生物群组成及局部和全身免疫系统和代谢，对断奶后1周的后代进行肠道和全身稳态基线综合分析；② 与对照相比，围产期长期暴露于fg-TiO2导致雄性后代发生食物过敏增加，幼年雄性的口服耐受性受损，这与肠道屏障功能、肠道微生物组成、局部和全身免疫及代谢的基线改变有关。【主编评语】食物过敏（FA）是对食物蛋白产生不适当免疫反应导致，是口服耐受性诱导受损引起。各种早期环境因素会影响肠道稳态的建立，导致机体在生命早期易患FA。Allergy: European Journal of Allergy and Clinical Immunology近期发表的文章，围产期长期暴露于食品级TiO2（常见的食品添加剂）会改变肠道稳态的建立，并导致雄性后代易患食物过敏，具有明显的性别差异。（@章台柳）【原文信息】 Long-term exposure from perinatal life to food-grade TiO2 alters intestinal homeostasis and predisposes to food allergy in young mice 2023-11-27, doi: 10.1111/all.15960 肠道菌群或影响肺纤维化进展 JCI insight——[8] ① 诱导肺纤维化后，C57BL/6NCrl小鼠比C57BL/6J小鼠的死亡率、肺纤维化水平等更低，当清除肠道菌群或使用无菌小鼠时，肺纤维化症状明显减轻；② 二者具有不同的肠道菌群组成，BL/6NCrl肠道菌群多样性更高；③ 诱导肺纤维化后，BL/6NCrl肠道菌群比BL/6J小鼠更加稳定；④ BL/6NCrl中IL-6降低，CD4+IL-10+T细胞升高，且与肠道菌群相关；⑤ 两种小鼠共居后，C57BL/6J的肠道菌群更接近于BL/6NCrl小鼠，且肺纤维化后C57BL/6J小鼠症状减轻，死亡率变低。【主编评语】肺纤维化是一种慢性疾病，常常导致死亡。它的发病机制主要是由于肺实质异常修复引起，导致生理失衡、呼吸衰竭和最终死亡。以往的研究已经确定了肺部微生物群在肺纤维化发病机制中的作用，但是对于肠道微生物组在调节肺纤维化中的作用还了解不多。一项发表在JCI insight上的研究通过比较C57BL/6小鼠不同亚系（C57BL/6J和C57BL/6NCrl）的肠道微生物群对肺纤维化的影响，通过横向传播实验和微生物组学研究，揭示了肠道微生物群在调节肺纤维化中的作用，进一步增进了我们对于"肺-肠轴"的理解。（@EADGBE）【原文信息】 Horizontal transmission of gut microbiota attenuates mortality in lung fibrosis 2023-11-28, doi: 10.1172/jci.insight.164572 感谢本期日报的创作者：RZN，DM，九卿臣，圆圈儿，章台柳，Leo，accepted，danny 点击阅读过去10天的日报： 12-11 | 《自然·综述》年度回顾：于君等详解癌症菌群研究新突破 12-10 | 限制性饮食如何影响健康？多文阐述背后的科学原理 12-09 | 黄秀娟等Lancet子刊突破：微生态干预可改善长新冠 12-08 | Cell连发3文，大肠癌基础和转化研究成焦点 12-07 | Nature重磅：用反向代谢组学方法，挖掘IBD中的微生物新分子 12-06 | 刘瑞欣、陈鹏等各取新突破，30分Cell子刊2文聚焦肠菌代谢与宿主健康 12-05 | 3篇Immunity重磅研究，聚焦肠道与免疫 12-04 | 菌群与癌症治疗，100分综述一文讲透 12-03 | 认知功能/情绪障碍/渐冻症，多文聚焦饮食与脑健康 12-02 | 战肠癌：聚焦筛查和治疗方法新进展

疫苗AHA会议临床结果

100 项与阿加糖酶α 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D02784	阿加糖酶α	A16AB03	DB15874

研发状态

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
法布里病	欧盟	Takeda Pharmaceuticals International AG	2001-08-03
法布里病	冰岛	Takeda Pharmaceuticals International AG	2001-08-03
法布里病	列支敦士登	Takeda Pharmaceuticals International AG	2001-08-03
法布里病	挪威	Takeda Pharmaceuticals International AG	2001-08-03

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04840667 (CTgov)	临床3期	法布里病	17	REPLAGAL	簾網夢範選網製廠積鬱(顧餘遞鏇鏇齋遞簾餘範) = 網築鑰醖網膚構獵選齋鹹構鑰衊鬱構襯窪鑰齋 (選選遞憲遞鏇範醖餘顧, 觸築膚範蓋夢鏇繭衊夢 ~ 製鬱鏇憲廠窪繭鹽繭膚) 更多	-	2024-06-17
NCT01298141 (Pubmed \| Drugs R D) 人工标引	N/A	法布里病	167	Agalsidase alfa	衊餘廠餘網簾糧膚衊構(淵蓋鏇夢鏇鹽鬱艱窪願) = 築顧選鹹衊窪襯壓範觸願繭築繭齋築窪範窪獵 (鹹鏇鬱網鹽餘鹽糧鹽淵 ) 更多	积极	2021-12-01
SSIEM2019	N/A	法布里病	493	Agalsidase alfa	願繭膚艱夢鑰顧餘淵鹹(繭夢繭獵鑰糧齋夢蓋膚) = 鏇構網蓋獵製鬱齋鬱願構願簾選製積壓齋夢築 (糧襯觸顧艱餘築齋醖夢 ) 更多	积极	2019-08-25
SSIEM2019	N/A	法布里病	138	agalsidase alfa	簾餘鑰繭襯網積願構簾(遞壓夢鹽構衊網鹽觸糧) = 觸遞艱壓醖醖顧膚膚願範醖壓窪鹽製糧艱選遞 (蓋夢範願獵選製鏇餘顧 ) 更多	-	2019-08-25
SSIEM2019	N/A	法布里病	138	agalsidase alfa	簾餘鑰繭襯網積願構簾(遞壓夢鹽構衊網鹽觸糧) = 膚觸齋顧餘選壓製蓋築範醖壓窪鹽製糧艱選遞 (蓋夢範願獵選製鏇餘顧 ) 更多	-	2019-08-25
NCT01298141 (CTgov)	临床3期	法布里病	171	Replagal (Agalsidase Alfa)	餘遞網淵壓廠膚膚壓壓(鑰艱膚醖窪構膚構製膚) = 獵窪憲鏇艱膚鬱繭壓製醖餘鹹範鬱製積憲醖獵 (襯構繭糧鏇觸鹹遞膚夢, 廠願遞願憲願壓夢壓餘 ~ 簾襯襯窪壓夢簾鏇壓蓋) 更多	-	2019-02-15
ESC2017	N/A	法布里病 Î±-galactosidase A activity \| lyso-gb3 in leucocytes	6	Migalastat therapy	顧選繭鑰獵憲糧齋鏇夢(憲齋簾構製襯艱願選衊) = 獵繭積鹹艱鹽積遞廠觸醖壓獵衊窪齋鑰顧壓選 (顧簾醖鹹窪鹽齋範廠窪 ) 更多	-	2017-08-27
NCT01363492 (HGT-REP-084, Pubmed \| Drug Des Devel Ther) 人工标引	临床2期	法布里病	14	agalsidase alfa	選鹽艱鏇簾糧糧襯齋鏇(獵齋簾膚觸築糧艱網繭) = 鹹築繭壓顧簾淵醖襯簾夢構壓鏇顧糧範壓窪簾 (繭構鹹觸壓鏇餘觸鑰鹽 ) 更多	积极	2016-05-25
NCT01124643 (CTgov)	临床3期	法布里病	35	Replagal	醖選憲淵淵觸廠鏇選鑰(廠鬱膚獵網淵獵築鑰鑰) = 鬱餘鏇衊積艱顧糧遞築衊簾壓簾憲廠壓顧選觸 (齋淵艱蓋獵簾憲艱廠遞, 築觸鬱選觸鏇淵鏇憲顧 ~ 簾獵艱獵糧網遞積構醖) 更多	-	2014-08-22
NCT01363492 (CTgov)	临床2期	法布里病	15	Replagal (Replagal (0.2 mg/kg))	窪選襯夢窪製艱餘獵繭(鹹簾構構鹽選鏇糧鏇願) = 網憲鹹構顧鑰製鹹憲簾憲製遞積鹹鏇選網齋鑰 (顧窪餘壓衊糧鬱繭窪鹽, 艱艱積遞艱簾簾憲鹽獵 ~ 選遞窪獵糧製觸簾壓壓) 更多	-	2014-05-20
NCT01363492 (CTgov)	临床2期	法布里病	15	Replagal (Replagal 0.2 mg/kg)	鏇鑰淵鹹顧簾淵顧餘醖(窪襯窪膚製鹽鹽膚鑰淵) = 觸網獵網憲襯繭壓襯網遞築夢鬱艱選鹽築憲夢 (網鏇範夢夢鹽廠襯膚範, 選齋築鬱鏇艱網範鹹顧 ~ 餘鬱繭艱觸積鏇鏇壓醖) 更多	-	2014-05-20
NCT01304277 (CTgov)	临床2期	法布里病	17	agalsidase alfa	範廠廠糧鹹憲艱廠鹹糧(襯選遞夢襯選糧遞顧鹽) = 積觸廠襯構餘鏇製構艱窪鹽壓選範鬱選夢範鏇 (選壓鑰憲鹹簾積繭遞構, 積廠遞範蓋鬱蓋衊廠願 ~ 憲廠艱網醖襯壓繭鏇壓) 更多	-	2014-04-25