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更新于：2025-05-07

Insulin-Like Growth Factor I Deficiency

胰岛素样生长因子I缺乏症

更新于：2025-05-07

基本信息

别名

GROWTH RETARDATION WITH SENSORINEURAL DEAFNESS AND MENTAL RETARDATION、Growth Retardation with Sensorineural Deafness and Mental Retardation、Growth delay due to insulin-like growth factor type 1 deficiency

+ [16]

简介

Insufficient circulating insulin-like growth factor-I.

关联

项与胰岛素样生长因子I缺乏症相关的药物

Mecasermin (Ipsen SA)

美卡舍明生（Ipsen SA）

靶点

IGF-1R

作用机制

IGF-1R激动剂

在研机构

Ipsen Biopharmaceuticals, Inc. [+2]

原研机构

Ipsen SA

在研适应症

成长不全 [+5]

非在研适应症

克罗恩病 [+4]

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2005-08-30

Mecasermin Rinfabate

美卡舍明林菲培

靶点

IGF-1R

作用机制

IGF-1R拮抗剂

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段临床2期

首次获批国家/地区

美国

首次获批日期2005-12-12

MOD-13012 (Opko Biologis)

靶点

IGF-1R

作用机制

IGF-1R激动剂

在研机构

Prolor Biotech Ltd.

原研机构

OPKO Biologics Ltd.

在研适应症

胰岛素样生长因子I缺乏症

非在研适应症-

最高研发阶段临床前

首次获批国家/地区-

首次获批日期1800-01-20

项与胰岛素样生长因子I缺乏症相关的临床试验

NCT05301569

/ RecruitingN/AIIT

Relation Between Serum Insulin Like Growth Factor 1 and Body Mass Index in Acne Vulgaris

cross-sectional study in which participants will be recruited at dermatology & Andrology out-patient clinics in Assuit university hospital, Egypt after patients examination by dermatologists & obtaining an informed consent

开始日期2023-04-01

申办/合作机构

Assiut University

NCT05642169

/ Unknown statusN/AIIT

Effect of a High Interval Intensity Training Program on Quality of Life, Mental Health, Brain Executive Functions, and IGF-1 Response in Sedentary Young University Women.

Sedentary lifestyle, understood as an activity that requires minimal body movement (Tremblay et al., 2017), is one of the main factors responsible for chronic diseases in Young adults.
In addition, this sedentary lifestyle generates mental disorders, such as anxiety, low self-esteem and depression, being more pronounced in women than in men (Nihill et al., 2013). Thus, both daily physical activity (PA) and physical exercise programs (PE), of moderate-vigorous intensity, act as an effective tools for the improvement of quality of life, since they generate benefits at physiological, psychological and social levels (Cohen et al., 2019).
If we focus on young adult, it can be seen how there is a significant decrease in the practice of physical exercise at this age (Grim et al., 2011). This means that the aforementioned recommendations are not reached (Cancela et al., 2019). Furthermore, if compared between sexes, lower levels are shown in the female sex (King et al., 2014).
For this reasons, and taking into account that the female population is a population vulnerable to significantly reduce their physical activity practice with age (Cohen et al., 2019), it is interesting to investigate on the possible health-related factors that this entails, such as quality of life, physical condition and mental health.
For these reasons, it is necessary to create effective strategies to address factors related to the main cognitive impairments and thus preserve better mental health.
Among all possible strategies, both pharmacological and non-pharmacological for the improvement of mental health, cognition and executive functions in young adults.Physical exercise has been shown to be a highly effective strategy at these ages (Heath et al., 2016). The stimulation of HIIT seems to reduce antioxidant responses. In recent years, there is a high interest in knowing the effect of HIIT on different health outcomes, such as physical and psychological fitness (Eather et al., 2019).
For all these reasons, sedentary lifestyles are an important public health factor associated with numerous pathologies and have been shown to have a significant cognitive involvement. Although we know that physical exercise can have a preventive role in the management of these associations, the conditioning factors of physical exercise are unknown, as well as the lifestyle factors that could contribute to a greater extent to the improvement of executive functionality in young women.

开始日期2022-10-15

申办/合作机构

University of Seville

NCT04710186

/ Unknown statusN/AIIT

The Influence of Androgen Receptor, Insulin Like Growth Factor 1 Receptor, and Insulin Receptor in Male Patients With Peripheral Artery Disease

Peripheral artery Disease (PAD) is a major cardiovascular disease that can impair quality of life (QoL). It affects more often male patients and its pathophysiology is not completely known. Probably androgen receptors and metabolism alteration may play an important role in the onset and in the progression of PAD towards its dreadful complications. The aim of this study is to evaluate the role of Androgen Recetpr , IGF-I receptor, and Insulin Receptor on the arterial wall of male patients with PAD undergoing open revascularization surgery.

开始日期2018-08-01

申办/合作机构-

100 项与胰岛素样生长因子I缺乏症相关的临床结果

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100 项与胰岛素样生长因子I缺乏症相关的转化医学

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0 项与胰岛素样生长因子I缺乏症相关的专利（医药）

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522

项与胰岛素样生长因子I缺乏症相关的文献（医药）

2025-03-24·Endocrinology

IGF1 Signaling Regulates Neuropeptide Expression in Hypothalamic Neurons Under Physiological and Pathological Conditions

Article

作者: Loganathan, Neruja ; Belsham, Denise D ; He, Wenyuan

AbstractInsulin-like growth factor 1 (IGF1) plays a critical role in metabolism and aging, but its role in the brain remains unclear. This study examined whether hypothalamic neurons respond to IGF1 and how its actions are modulated. RT-qPCR and single-cell RNA sequencing indicated that Igf1r mRNA is expressed in neuropeptide Y/Agouti-related peptide (NPY/AgRP) neurons but has higher expression in pro-opiomelanocortin (POMC) neurons. IGF1 binding proteins Igfbp3 and Igfbp5 were significantly expressed, whereby Igfbp5 levels were modulated by fasting, nutrient availability, and circadian rhythms, implying that IGF1 signaling can be controlled by multiple mechanisms. In mouse and human models, IGF1 regulated Agrp, Npy, Pomc, Cartpt, Spx, Gal, and Fam237b expression, producing an overall anorexigenic profile. Hyperinsulinemia induced IGF1 resistance, accompanied by reduced IGF1R protein, as well as Igf1r and Irs2 mRNA expression via over-activation of phosphoinositide 3-kinase/forkhead box O1 (PI3K-FOXO1) signaling. Thus, hypothalamic neurons respond to IGF1 under physiological conditions, and hyperinsulinemia is a novel mechanism that drives cellular IGF1 resistance.

2025-02-01·Biomedicine & Pharmacotherapy

IGF1 enhances memory function in obese mice and stabilizes the neural structure under insulin resistance via AKT-GSK3β-BDNF signaling

Article

作者: Jo, Danbi ; Song, Juhyun ; Choi, Seo Yoon ; Ahn, Seo Yeon

Obesity is a prevalent metabolic disorder linked to insulin resistance, hyperglycemia, increased adiposity, chronic inflammation, and cognitive dysfunction. Recent research has focused on developing therapeutic strategies to mitigate cognitive impairment associated with obesity. Insulin growth factor-1 (IGF1) deficiency is linked to insulin resistance, glucose intolerance, and the progression of obesity-related central nervous system (CNS) disorders. In this study, we investigated the neuroprotective effects of IGF1 in two obesity models: diet-induced obesity (high-fat diet mice) and genetic obesity (ob/ob mice which is genetically deficient in leptin), and in vitro Neuro2A neuronal cells and primary cortical neurons under insulin resistance conditions. We performed RNA sequencing analysis using the cortex of high-fat diet mice injected with IGF1. Also, we detected cytokine levels in blood of high-fat diet mice injected with IGF1. In addition, we conducted the Barnes maze test as a spatial memory function test and open field test as an anxiety behavior test in ob/ob mice. We measured the levels of proteins and mRNAs related to insulin signaling, including synaptic density proteins in brain cortex of ob/ob mice. Our results showed that IGF1 injection enhanced spatial memory function and synaptic plasticity in obese mice. Furthermore, in vitro data demonstrated that IGF1 treated neurons revealed enhanced neural complexity and improved neurite outgrowth under insulin resistance condition through the AKT-GSK3β-BDNF pathway related to antidepressant, cognitive function and anti-apoptotic mechanisms. Therefore, our results provided that IGF1 have potential to alleviate cognitive impairment by promoting synaptic plasticity and neural complexity in the obese brain.

2025-01-01·Neurotherapeutics

IGF-1 impacts neocortical interneuron connectivity in epileptic spasm generation and resolution

Article

作者: Le, John T ; Ballester-Rosado, Carlos J. ; Lam, Trang T. ; Le, John T. ; Frost, James D. ; Ballester-Rosado, Carlos J ; Anderson, Anne E. ; Swann, John W. ; Anderson, Anne E ; Swann, John W ; Lam, Trang T ; Frost, James D

Little is known about the mechanisms that generate epileptic spasms following perinatal brain injury. Recent studies have implicated reduced levels of Insulin-like Growth Factor 1 (IGF-1) in these patients' brains. Other studies have reported low levels of the inhibitory neurotransmitter, GABA. In the TTX brain injury model of epileptic spasms, we undertook experiments to evaluate the impact of IGF-1 deficiencies on neocortical interneurons and their role in spasms. Quantitative immunohistochemical analyses revealed that neocortical interneurons that express glutamic acid decarboxylase, parvalbumin, or synaptotagmin 2 co-express IGF-1. In epileptic rats, expression of these three interneuron markers were reduced in the neocortex. IGF-1 expression was also reduced, but surprisingly this loss was confined to interneurons. Interneuron connectivity was reduced in tandem with IGF-1 deficiencies. Similar changes were observed in surgically resected neocortex from infantile epileptic spasms syndrome (IESS) patients. To evaluate the impact of IGF-1 deficiencies on interneuron development, IGF-1R levels were reduced in the neocortex of neonatal conditional IGF-1R knock out mice by viral injections. Four weeks later, this experimental maneuver resulted in similar reductions in interneuron connectivity. Treatment with the IGF-1 derived tripeptide, (1-3)IGF-1, abolished epileptic spasms in most animals, rescued interneuron connectivity, and restored neocortical levels of IGF-1. Our results implicate interneuron IGF-1 deficiencies, possibly impaired autocrine IGF-1 signaling and a resultant interneuron dysmaturation in epileptic spasm generation. By restoring IGF-1 levels, (1-3)IGF-1 likely suppresses spasms by rescuing interneuron connectivity. Results point to (1-3)IGF-1 and its analogues as potential novel disease-modifying therapies for this neurodevelopmental disorder.

项与胰岛素样生长因子I缺乏症相关的新闻（医药）

2025-04-02

·PipelineReview

ESTEVE signs a licensing agreement for a severe primary insulin-like growth factor 1 deficiency treatment

BARCELONA, Spain I April 1, 2025 I ESTEVE has signed a license and supply agreement with Eton Pharmaceuticals with global rights for Increlex®, except for USA. This biologic product, the first biologic in ESTEVE’s portfolio, is used for the long-term treatment of growth failure in patients aged 2 to 18 years due to a condition known as ‘severe primary insulin-like-growth-factor-1 deficiency’ (SPIGFD). Patients with this condition have low levels of the hormone insulin-like growth factor-1 or IGF-1, which is required for normal growth. 1 “As a rare disorder, patients suffering SPIGFD, often face difficulties associated with receiving a late correct diagnosis, with consequent delayed treatment initiation with limited access to appropriate therapy. This has a considerable impact on the physical health and quality of life of these patients. 2 The disease awareness and the multidisciplinary approach are critical to improve those people’s lives. This standard of care medicine is perfectly aligned with our strategy,focused on highly specialized treatments for high unmet medical and patient needs”, confirms José María Giménez Arnau, Chief Scientific & Medical Officer of ESTEVE. Increlex® was designated an ‘orphan medicine’ (a medicine used in rare diseases) in 2006. 1 It is approved by the U.S. Food and Drug Administration (FDA) 3 and European Medicines Agency (EMA) 1 and authorized in 40 territories. References About ESTEVE ESTEVE ( www.esteve.com ) is a global pharmaceutical company with a clear purpose: to improve people’s lives. Founded in 1929 and headquartered in Barcelona, ESTEVE has a strong international presence with pharmaceutical affiliates in Spain, Portugal, Italy, Germany, France, the UK, and the USA. ESTEVE is focused on delivering highly specialized treatments that address significant unmet medical needs in several therapeutic areas. In addition, to our innovative pharma business, we offer comprehensive Contract Manufacturing services (CMO), specializing in the production of Active Pharmaceutical Ingredients (APIs) through world-class facilities in Spain, Mexico, and China. ESTEVE’s strong commitment to its core values—people matter, transparency, and accountability—remains at the heart of everything it does. SOURCE: Esteve

引进/卖出孤儿药上市批准

2025-04-01

·PRNewswire

ESTEVE signs a licensing agreement for a severe primary insulin-like growth factor 1 deficiency treatment

It is a biologic product used to treat children and adolescents from 2 to 18 years-old who suffer from severe primary insulin-like growth factor 1 deficiency1 The acquisition of the treatment for this rare disease is an example of ESTEVE's commitment focused on growth opportunities in high unmet medical and patient needs. BARCELONA, Spain, April 1, 2025 /PRNewswire/ -- ESTEVE has signed a license and supply agreement with Eton Pharmaceuticals with global rights for Increlex®, except for USA. This biologic product, the first biologic in ESTEVE's portfolio, is used for the long-term treatment of growth failure in patients aged 2 to 18 years due to a condition known as 'severe primary insulin-like-growth-factor-1 deficiency' (SPIGFD). Patients with this condition have low levels of the hormone insulin-like growth factor-1 or IGF-1, which is required for normal growth.1 "As a rare disorder, patients suffering SPIGFD, often face difficulties associated with receiving a late correct diagnosis, with consequent delayed treatment initiation with limited access to appropriate therapy. This has a considerable impact on the physical health and quality of life of these patients.2 The disease awareness and the multidisciplinary approach are critical to improve those people's lives. This standard of care medicine is perfectly aligned with our strategy,focused on highly specialized treatments for high unmet medical and patient needs", confirms José María Giménez Arnau, Chief Scientific & Medical Officer of ESTEVE. Increlex® was designated an 'orphan medicine' (a medicine used in rare diseases) in 2006.1 It is approved by the U.S. Food and Drug Administration (FDA)3 and European Medicines Agency (EMA)1 and authorized in 40 territories. References Increlex | European Medicines Agency (EMA) Challenges in the care of individuals with severe primary insulin-like growth factor-I deficiency (SPIGFD): an international, multi-stakeholder perspective - PubMed Drug Approval Package: Increlex (Mecasermin [rDNA origin]) NDA #021839 About ESTEVE ESTEVE () is a global pharmaceutical company with a clear purpose: to improve people's lives. Founded in 1929 and headquartered in Barcelona, ESTEVE has a strong international presence with pharmaceutical affiliates in Spain, Portugal, Italy, Germany, France, the UK, and the USA.   ESTEVE is focused on delivering highly specialized treatments that address significant unmet medical needs in several therapeutic areas. In addition, to our innovative pharma business, we offer comprehensive Contract Manufacturing services (CMO), specializing in the production of Active Pharmaceutical Ingredients (APIs) through world-class facilities in Spain, Mexico, and China.  ESTEVE's strong commitment to its core values—people matter, transparency, and accountability—remains at the heart of everything it does.  For more information: Irene Simón, Head of Global External Communications & ESG, ESTEVE Tel.+34 934 466 000 – [email protected] Gemma Mestre, External & Digital Communications Manager, ESTEVE Tel.+34 934 466 000 – [email protected] Logo - SOURCE ESTEVE WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

引进/卖出孤儿药

2025-02-28

·金赛药业

国际罕见病日——为每一个生命，全力以赴

为每一个生命，全力以赴今天是第18个国际罕见病日，关爱罕见病患者群体，让生命不“罕”见，让所有人被世界温柔对待，是医药人矢志追寻的初心。作为妇女儿童健康领域的守护者，金赛药业积极响应国家罕见病药物研发号召，把目光投向妇女与儿童罕见病领域，基于公司在基因工程领域的创新技术和产业化优势，将重组人生长激素应用于罕见病临床诊疗，积极开展患者关爱活动，提升罕见病治疗可及性。看见“隐形”之痛：妇儿罕见病亟待破局根据《中国罕见病定义研究报告2021》的定义，罕见病为新生儿发病率小于万分之一、患病率小于万分之一、患病人数小于14万的疾病。在全球约3.5亿罕见病患者中，妇女与儿童占比超过60%。她们往往面临更复杂的困境：孕期罕见病误诊导致母婴双重风险、儿童发育类罕见病因认知不足错过黄金干预期、女性患者承受社会角色与疾病的双重压力……这些“生命难题”背后，是诊断技术滞后、药物可及性低、社会支持体系薄弱的行业现状。他们中的许多人，终其一生都在以超乎常人的勇气与疾病抗争，在等待希望，而守护这份生命的希望，正是医药人刻在基因里的使命。金赛药业深耕儿童健康领域二十余年，针对临床未被满足的罕见病诊疗需求，公司持续投入罕见病药物开发。通过对生长发育类罕见病的研究，不断加深对重组人生长激素这一罕见病用药的理解，如小胖威利综合征、特纳综合征、努南综合征、软骨发育不全和原发性生长激素缺乏症引起的儿童生长发育障碍等罕见病，已被纳入生长激素的适应症范围，越来越多的罕见病患儿开始通过生长激素治疗获益。以科学为炬：做“不可能”的破壁者面对罕见病研发高投入、高风险、低回报的行业难题，金赛选择以长期主义践行承诺。目前，研发管线涵盖国家罕见病目录中原发性胰岛素样生长因子-1缺乏症、全身型特发性关节炎和Silver-Russell综合征等儿童生长发育障碍相关病症。同时公司还布局了针对囊性纤维化、苯丙酮尿症和有机酸血症等罕见病的创新管线，以期为全球罕见病患者提供更多的治疗药物选择。以责任为锚：构建有温度的生命支持网络药物研发是起点而非终点，持续推进罕见病临床研究的同时，公司还积极支持罕见病学术组织，协助中华医学会儿科学分会内分泌遗传代谢学组成立中国普拉德威利综合征(PWS)临床规范化多学科诊疗协作联盟，以及Silver-Russell综合征诊治协作组成立。金赛支持国际罕见病日患者关爱活动践行社会责任，公司还积极推进社会公益项目，构建有温度的罕见病患者生命支持网络。为帮助罕见病患者改善生活质量，减轻困难家庭的经济压力，2018年以来，金赛药业公益支持中国红十字基金会成长天使基金“小胖威利患者关爱项目”启动，为全国2周岁以内新确诊小胖威利患者提供医疗救助，支持开展小胖威利疾病相关基层医护人员培训、科普教育、科学研究等活动，目前，项目已救助700余人次。2021年，公司与成长天使基金联合全国儿童特纳综合征诊疗协作组共同启动“特纳综合征爱心救助项目”，为100名家庭经济困难的特纳综合征患者，免费提供一年生长激素药品资助，给患病女童送去健康关怀和成长希望。2023年至今，金赛已连续三年全程公益支持中国红十字基金会成长天使基金举办“为爱呐罕·让爱不凡”国际罕见病日患者关爱活动（上图）。疾病或许罕见，但爱与希望并不独行。在国际罕见病日这个特殊时刻，金赛药业愿以科学之翼守护每一个与众不同的生命，让更多患者及时得到科学医治，让罕见被看见。金赛药业为每一个生命，全力以赴！ END

核酸药物基因疗法