更新于：2024-05-01

Diabetes Insipidus

尿崩症

更新于：2024-05-01

基本信息

别名

DI、DI - Diabetes insipidus、DIABETES INSIPIDUS

+ [10]

简介

A disease that is characterized by frequent urination, excretion of large amounts of dilute URINE, and excessive THIRST. Etiologies of diabetes insipidus include deficiency of antidiuretic hormone (also known as ADH or VASOPRESSIN) secreted by the NEUROHYPOPHYSIS, impaired KIDNEY response to ADH, and impaired hypothalamic regulation of thirst.

关联

项与尿崩症相关的药物

CBT-125

靶点-

作用机制-

在研机构

Cipla BioTec Ltd.

原研机构

Cipla BioTec Ltd.

在研适应症

尿崩症 [+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

印度

首次获批日期2016-04-30

Desmopressin Acetate

醋酸去氨加压素

靶点

AVPR2

作用机制

AVPR2激动剂

在研机构

Ferring Pharmaceuticals, Inc. [+1]

原研机构

Ferring BV

在研适应症

尿崩症 [+2]

非在研适应症

乳腺癌 [+7]

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1978-02-21

Bendroflumethiazide

苄氟噻嗪

靶点

NCC

作用机制

NCC抑制剂

在研机构

天津世纪药业有限公司

原研机构

Bristol Myers Squibb Co.

在研适应症

尿崩症 [+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1959-12-07

项与尿崩症相关的临床试验

ACTRN12624000191594 / Not yet recruitingN/AIIT

Investigating tolvaptan stimulated copeptin measurements for the differential diagnosis of polyuria-polydipsia syndrome

开始日期2024-05-01

申办/合作机构-

CTR20234084 / CompletedN/A

醋酸去氨加压素片（0.1 mg）空腹/餐后状态下在健康受试者中的生物等效性试验

主要试验目的：在健康男性与女性受试者中于空腹/餐后条件下，评价受试制剂醋酸去氨加压素片（规格：0.1 mg，生产商：江苏利泰尔药业有限公司）和辉凌国际制药（瑞士）有限公司生产的参比制剂醋酸去氨加压素片（弥凝®，规格：0.1 mg）的生物等效性。

开始日期2024-01-12

申办/合作机构

江苏利泰尔药业有限公司

NCT06036004 / Recruiting临床2期

Oxytocin Substitution Therapy in Patients With Central Diabetes Insipidus: a Double-blind Randomised Placebo-controlled Trial: the OxyTUTION Trial

This randomized, placebo-controlled, double-blind trial aims to investigate intranasal OXT as a novel therapeutical option in central diabetes insipidus (cDI) to improve psychological symptoms and socio-emotional functioning.
Optionally, patients can present for additional assessments in sub-studies:
fMRI sub-study at day 14 (± 2 days) (one additional visit)
Social-stress sub-study at day 14 (± 2 days) (one additional visit)

开始日期2024-01-08

申办/合作机构

Universitätsspital Basel

100 项与尿崩症相关的临床结果

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100 项与尿崩症相关的转化医学

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0 项与尿崩症相关的专利（医药）

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15,649

项与尿崩症相关的文献（医药）

2024-09-30·Acta neurologica Taiwanica

Hyperintensity in Bilateral Posterior Limbs of Internal Capsule Due to Hypernatremia.

Article

作者: Min-Yang Wu ; Yuan-Ting Sun

PURPOSE:

Osmotic demyelination syndrome is usually caused by rapid correction of hyponatremia but sometimes develops from acute severe hypernatremia. Studies suggested that serum sodium increasing at the rate of less than 6~8 mmol/L in 24 hours has a low risk of osmotic demyelination syndrome, but sometimes exceptions present. Aside from the classical sites of involvement, such as pons and basal ganglia, internal capsules are rarely affected. We report a case with acute paraparesis caused by acute hypernatremia-induced extrapontine myelinolysis involving the posterior limbs of bilateral internal capsules.

CASE:

A 54-year-old man was admitted for aseptic encephalitis and moved to the intensive care unit due to poor consciousness and respiratory failure. Although cerebrospinal fluid pleocytosis was improved later, acute hypernatremia due to partial diabetes insipidus developed. He presented acute paraplegia two days later with a negative result on the whole spine MRI. Although the increasing rate of serum sodium did not exceed the recommended safety range, the extrapontine myelinolysis involving posterior limbs of the bilateral internal capsule, as long as the corpus callosum, still developed. The patient regained partial walking ability after one year.

CONCLUSION:

This case report extends the spectrum of classical osmotic demyelination in clinical manifestations, image findings, and the causal range of electrolyte derangements.

2024-04-01·Arthroscopy, sports medicine, and rehabilitation

Tibial Tubercle Trochlear Groove Distance Does Not Correlate With Patellar Tendon Length in Patients Who Underwent Anterior Cruciate Ligament Reconstruction.

Article

作者: Emma E Johnson ; William L Johns ; Bryson Kemler ; Rahul Muchintala ; Ryan W Paul ; Manoj Reddy ; Brandon J Erickson

Purpose:

To examine the relationship between tibial tubercle-trochlear groove (TT-TG) distance and patellar tendon length.

Methods:

All healthy athletes who underwent anterior cruciate ligament reconstruction who had a magnetic resonance imaging (MRI) study of the knee on file between July 2018 and June 2019 at a single institution were retrospectively reviewed. Exclusion criteria included patients without an MRI study of the knee on file or with an MRI of insufficient quality precluding reliable calculation of TT-TG and patellar tendon length. MRIs were reviewed to calculate TT-TG, patellar tendon length, and Caton-Deschamps Index (CDI). Patient charts were reviewed to obtain anthropometric characteristics including sex, concomitant injuries, and previous knee procedures as well as age at time of MRI. Spearman correlations were used to assess the relationship between TT-TG, patellar tendon length, and CDI, with regression analysis performed to assess for relationships between TT-TG, patellar tendon length, and patient-specific factors.

Results:

Overall, 235 patients (99 female [42.1%], 136 male [57.9%]; mean age: 30.0 years [23.0; 40.0]) were included. Inter-rater reliability between the 2 reviewers was 0.888 for TT-TG, 0.804 for patellar tendon length, and 0.748 for CDI, indicating strong agreement. The correlation between TT-TG and patellar tendon length was 0.021, indicating no true relationship. The correlation between TT-TG and CDI was -0.048 and that of patellar tendon length and CDI was 0.411, indicating a weak positive relationship. Regression analysis found that male sex is strongly correlated with a longer patellar tendon length (odds ratio 2.65, 95% confidence interval 1.33-3.97, P < .001).

Conclusions:

In this study, no correlation was found between TT-TG and patellar tendon length or CDI. Male sex was correlated with a longer patellar length.

Level of Evidence:

Level III.

2024-02-29·Heliyon

Neuro-ophthalmological findings of pituitary metastasis: Case series from a single center and review of the literature.

Review

作者: Hongliang Wang ; Mingjie Zhu ; Yan Yan

When pituitary metastasis (PM) invades the pituitary gland, it leads to impaired endocrine function and compression and infiltration of surrounding tissues, causing a series of clinical alterations. We presented seven patients with PM evaluated at neuro-ophthalmologic clinic and reviewed neuro-ophthalmological findings of 44 cases with PM in the English literature from 1979 to December 2022. The mean age at diagnosis was 58.1 years, and 45.1% were male. The mean latency period from primary cancer to the diagnosis of PM was 57.6 months. PM is the only presenting sign of malignancy in 11.8% of patients. The mean time from the visual disturbance to the diagnosis of PM was 96.3 days. Visual acuity decreased in 72.5% and 60.8% of cases with visual field defects. 74.2% exhibited a pattern of temporal field defect respecting the vertical meridian. Isolated ophthalmoplegia was found in 37.3% of patients. The most common ocular motor nerve palsy was unilateral III nerve palsy. Breast cancer was the most common primary malignancy. 84.6% entirely or partially relieved the neuro-ophthalmic symptoms after treatment. 51% of patients were alive during a mean follow-up period of 11 months. The mean survival duration was within six months in 65% of deceased patients. For elderly patients with a pituitary tumor, PM should be on the list of differential diagnoses for those with visual impairment, especially ocular motor nerve palsy, even if diabetic insipidus is not present, with or even without a history of malignancy, regardless of the primary tumor site.

268

项与尿崩症相关的新闻（医药）

2024-04-05

·药事纵横

3期临床失败，Amylyx渐冻症药主动撤市

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。4月4日，Amylyx Pharmaceuticals宣布与美国食品和药物管理局（FDA）和加拿大卫生部达成协议，主动撤市治疗肌萎缩侧索硬化（ALS）成人患者的药物Relyvrio（AMX0035）。作出这一决定的主要原因是其三期临床PHOENIX的顶线数据，研究没有达到主要终点和次要终点。PHOENIX研究共招募了664例ALS患者，结果并未达到主要终点，第48周时，与安慰剂组比，AMX0035治疗组肌萎缩侧索硬化功能评定量表（ALSFRS-R）评分较基线变化不具有显著统计学差异（p=0.667）。此外，该研究也未达到患者报告的生活质量结果评估、总生存期以及慢肺活量（SVC）衡量的呼吸功能等次要终点。Relyvrio将不再适用于新患者。目前正在美国和加拿大接受治疗的患者，在咨询他们的医生后，希望继续治疗，可以过渡到免费的药物计划。在研管线作为公司发现和开发神经退行性疾病的创新治疗方案的一部分，Amylyx将继续推进两个关键项目，研究其用于Wolfram综合征和进行性核上性麻痹(PSP)的候选药物AMX0035，以及用于ALS的靶向calpain-2的反义寡核苷酸AMX0114。Amylyx 首席医疗官 Camille L. Bedrosian 医学博士说，我们将继续关注ALS，相信AMX0114在治疗ALS和其他疾病方面具有强大的潜力。钙离子依赖的中性蛋白酶-I2被认为是轴突变性过程中的关键蛋白，在已发表的研究中多次与神经丝生物学联系在一起。在我们对AMX0114的临床前研究和多个独立发表的研究中，抑制钙离子依赖的中性蛋白酶-I 2减少了细胞死亡和变性，降低了神经丝水平。我们期待在今年下半年启动AMX0114在ALS中的临床试验。此外，Amylyx还宣布将进行重组，将公司的财务资源集中在即将到来的临床里程碑上，该公司将裁员约70%。随着此次计划的开展，Amylyx预计现金跑道将延长至2026年，将专注于关键里程碑包括HELIOS (AMX0035治疗Wolfram综合征)、ORION (AMX0035治疗PSP)的临床数据，以及AMX0114治疗ALS的计划试验。Amylyx Pharmaceuticals成立于2013年，是一家总部位于马萨诸塞州剑桥市的制药公司，致力于开发神经退行性疾病的潜在疗法。大约十年前，两位联合创始人 Justin Klee 和 Joshua Cohen 在还是布朗大学的本科生时构思了 Relyvrio。他们的想法是，将牛磺二醇（一种有时用于调节肝酶的补充剂）和苯丁酸钠（一种治疗儿童尿素紊乱的药物）结合起来，可以通过防止细胞中两种结构（线粒体和内质网）的功能障碍，保护大脑中的神经元免受 ALS 等疾病的损害。联合创始人Justin Klee（右）和 Joshua Cohen（左）作为唯一在售产品，Relyvrio 在 2023 年为公司带来了 3.808 亿美元的产品净收入。截至 2023 年底，该公司拥有现金 3.714 亿美元。其 2020 年的运营费用为 3970 万美元，2023 年为 3.42 亿美元。参考资料：www.amylyx.comwww.endpts.com-END-药事纵横投稿须知：稿费已上调，欢迎投稿各位朋友好，觉得本文对您有帮助，请随手点一下下方的在看，以便让你的朋友也能看到哦。

临床3期引进/卖出临床失败临床2期寡核苷酸

2024-04-04

·Firstwordpharma

Amylyx to withdraw ALS therapy Relyvrio in US, Canada

Amylyx Pharmaceuticals will withdraw Relyvrio (sodium phenylbutyrate/taurursodiol) from the US market, following through on a commitment made when the amyotrophic lateral sclerosis (ALS) therapy controversially won accelerated approval in 2022. At that time, joint CEO Justin Klee said such a move would be likely if a confirmatory study failed – the company announced last month that the PHOENIX trial was a dud.Klee, along with joint CEO Joshua Cohen, said Thursday that the decision to withdraw Relyvrio was “reached…in partnership with the stakeholders who will be impacted.” Amylyx, which will also pull the drug in Canada, where it is sold as Albrioza, noted that it will provide the therapy free of charge for those currently on treatment who want to continue.Staff losses of 70%The drugmaker will also cut around 70% of its workforce as part of a restructuring plan as it looks to prioritise resources, extending its cash runway into 2026. Amylyx is also investigating the oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol - known as AMX0035 - in Wolfram syndrome and progressive supranuclear palsy (PSP). Interim data from the Phase II HELIOS trial of AMX0035 for the treatment of Wolfram syndrome are expected later this month.Meanwhile, chief medical officer Camille Bedrosian, noted that the Phase III ORION study of AMX0035 for PSP is ongoing, with “an interim analysis…now expected in mid-2025.” Amylyx has also not given up on ALS, with AMX0114, an antisense oligonucleotide targeting calpain-2, set to enter clinical studies in the second half of the year.For related analysis, read KOL Views Q&A: Leading neurologist outlines where ALS field goes next after Amylyx’s Relyvrio failure.

临床2期临床3期寡核苷酸上市批准

2024-04-04

·BioSpace

Amylyx to Pull ALS Drug Relyvrio From US, Canada Markets After Phase III Fail

Pictured: Illustration showing a neuron being destroyed/iStock, Dr_Microbe Amylyx Pharmaceuticals on Thursday announced that it has started the process of pulling its amyotrophic lateral sclerosis drug Relyvrio (sodium phenylbutyrate and taurursodiol) from the U.S. and Canadian markets and will reduce the company’s workforce by approximately 70% under a restructuring plan. Effective Thursday, Relyvrio will no longer be available for new patients. Those who are currently being treated with Relyvrio can either stay on treatment or transition into a free program, depending on their physician’s advice, according to the company. Amylyx said it will work with the FDA and Health Canada to terminate the marketing authorizations for Relyvrio in these countries. Joshua Cohen and Justin Klee, co-CEOs of Amylyx, in a statement called the withdrawal of Relyvrio a “difficult moment” for the company and for patients with amyotrophic lateral sclerosis (ALS). “The decision to remove Relyvrio from the market and provide therapy free of charge for those who wish to continue was informed by the PHOENIX trial results, engagement with regulatory authorities, and discussions with the ALS community,” Cohen and Keel said. Last month, Relyvrio failed its highly anticipated Phase III PHOENIX trial, unable to significantly improve scores in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale at 48 weeks versus placebo. Amylyx’s drug also fell short of its secondary endpoints, including slow vital capacity and self-reported health status, as measured by the Amyotrophic Lateral Sclerosis Assessment Questionnaire. Amylyx will present data from PHOENIX at the upcoming American Academy of Neurology meeting, according to Thursday’s announcement. The study’s open-label extension phase is also ongoing, and Amylyx plans to collect survival data from the trial to help inform future studies in ALS. Losing its only commercial product, Amylyx on Thursday also launched a sweeping restructuring initiative to cut costs and optimize the use of its resources, with an eye toward upcoming clinical milestones. The biotech will lay off around 70% of its employees and lower financial commitments outside of its new priority areas. Amylyx will now focus on its lead asset AMX0035—which, like Relyvrio, also contains sodium phenylbutyrate and taurursodiol—being developed for Wolfram syndrome and progressive supranuclear palsy. The biotech is also advancing the antisense oligonucleotide candidate AMX0114 in ALS. CMO Camille Bedrosian revealed in a statement that Amylyx will present Phase II data for AMX0035 in Wolfram syndrome later this month while its Phase III ORION study in progressive supranuclear palsy is set for an interim analysis in mid-2025. The biotech is also preparing to initiate a clinical trial for AMX0114 in the second half of 2024. The company restructuring and priority changes should extend Amylyx’s cash runway into 2026, according to the announcement. 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3期寡核苷酸临床2期