Prospective, Multicentre Study to Validate the GastroIntestinal Dysfunction Score (GIDS) and Describe Prevalence, Outcomes, and Management of Phosphate Disorders in Intensive Care Patients

Part A Multiple organ dysfunction syndrome is one of the main problems in critically ill patients. Several organ dysfunctions are routinely and systematically monitored in patients admitted to intensive care units. Gastrointestinal dysfunction is currently not included in multiple organ dysfunction scores and lacks a standardised approach. At the same time, investigators earlier studies have shown that gastrointestinal dysfunction plays an essential role in the course of illness in intensive care patients. A tool for measuring dysfunction is needed to enable future studies on interventions to improve gastrointestinal function. The GIDS (GastroIntestinal Dysfunction Score) combines different clinical signs and symptoms commonly observed and documented in patients in the ICU into a scoring system, allowing distinguishing between different grades of severity of dysfunction. The investigators will only use observations documented during the patient's stay in the intensive care unit without additional measurements and calculate gastrointestinal (GIDS) and other organ dysfunction scores.
Part B Phosphate is an electrolyte commonly measured and often corrected, while the indications and clear guidance for correction are insufficiently studied. The results of this study will assist in specifying indications for the correction of phosphate levels better and refine the management strategies in the future. Only phosphate levels measured as part of routine care will be documented, no additional samples for study purposes will be taken, and the study will not influence the treatment of phosphate disorders.

开始日期2024-05-01

申办/合作机构

University Hospitals of North Midlands NHS Trust

NCT06015750 / Not yet recruiting临床4期

An Interventional, Prospective Open-Label Study of Immunosuppressive Therapies to Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia (RESTORE)

The primary objective of this study is to evaluate the effect of immunosuppressive therapy (IST) in participants treated with asfotase alfa who demonstrate immune-mediated loss of effectiveness (LoE).

开始日期2024-04-30

申办/合作机构

Alexion Pharmaceuticals, Inc.

NCT06079372 / Recruiting临床3期

A Phase 3, Randomized, Open-label, Parallel-arm, Active-controlled, Multicenter Study to Evaluate Safety and Efficacy of ALXN1850 Versus Asfotase Alfa Administered Subcutaneously in Pediatric Participants (2 to < 12 Years of Age) With Hypophosphatasia (HPP) Previously Treated With Asfotase Alfa

The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.

开始日期2024-04-02

申办/合作机构

Alexion Pharmaceuticals, Inc.

100 项与金属代谢病相关的临床结果

登录后查看更多信息

100 项与金属代谢病相关的转化医学

登录后查看更多信息

0 项与金属代谢病相关的专利（医药）

登录后查看更多信息

34,710

项与金属代谢病相关的文献（医药）

2024-12-01·Renal failure

Rare renal proximal tubular dysfunctions in primary biliary cholangitis.

Article

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

INTRODUCTION:

Renal 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.

METHODS:

We identified 11 cases of PBC with PT dysfunctions (PBC-PT). Their medical document, kidney pathology, and follow-up data were retrospectively reviewed and analyzed.

RESULTS:

The 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).

CONCLUSIONS:

The PT dysfunctions were rare in PBC patients, and glucocorticoids treatment could benefit the improvements of eGFR and tubular functions.

2024-06-01·IBRO neuroscience reports

Correlation between 25-hydroxy-vitamin D and Parkinson's disease.

Article

作者: Ming Xia ; Qingjiu Zhou

Background:

Previous cross-sectional studies have shown that Parkinson's disease (PD) patients have lower serum 25-hydroxyvitamin D (25(OH)D) concentrations than controls. Other studies have not yet tested whether research findings from other regions are generalizable to Chinese populations. In this case-control study, we examined the correlation between 25-hydroxyvitamin D and Parkinson's disease.

Methods:

We established an association between 25-hydroxyvitamin D deficiency and PD in a case-control study of 100 PD patients and 100 control subjects free of neurological disease at the First Affiliated Hospital of Xinjiang Medical University.

Results:

Total 25-hydroxyvitamin D levels were deficient in 21 % of patients with PD compared with 4 % of controls. In univariate analyses, plasma levels of 25-hydroxyvitamin D were associated with PD (p < 0.001). In multivariate analyses, vitamin D deficiency (25(OH)D < 20 ng/mL) was significantly associated with PD (p = 0.008, Odds Ratio =17.13, 95 % CI= 2.082-141.075). Individuals with 25(OH)D levels in the lowest quartile had the highest prevalence of PD (p = 0.026, OR=11.786, 95 % CI =1.342-103.51 compared to individuals with values in the highest quartile).

Conclusions:

Our study reveals an association between 25-hydroxyvitamin D and PD. Patients with incident PD had significantly lower serum 25(OH)D concentrations than age-matched controls. High-risk PD patients with vitamin D deficiency who have not yet developed exercise impairment should undergo vitamin D measurement and any necessary treatment as soon as possible. Limitations of the study: the study needs further assessment of populations with low vitamin D levels in other regions of China; further assessment of the effect of different sources of vitamin D on PD; further study of longitudinal cohorts at different time points.

2024-03-01·Journal of cell science

Regulation of the apico-basolateral trafficking polarity of the homologous copper-ATPases ATP7A and ATP7B.

Article

作者: Ruturaj ; Monalisa Mishra ; Soumyendu Saha ; Saptarshi Maji ; Enrique Rodriguez-Boulan ; Ryan Schreiner ; Arnab Gupta

The homologous P-type copper-ATPases (Cu-ATPases) ATP7A and ATP7B are the key regulators of copper homeostasis in mammalian cells. In polarized epithelia, upon copper treatment, ATP7A and ATP7B traffic from the trans-Golgi network (TGN) to basolateral and apical membranes, respectively. We characterized the sorting pathways of Cu-ATPases between TGN and the plasma membrane and identified the machinery involved. ATP7A and ATP7B reside on distinct domains of TGN in limiting copper conditions, and in high copper, ATP7A traffics to basolateral membrane, whereas ATP7B traverses common recycling, apical sorting and apical recycling endosomes en route to apical membrane. Mass spectrometry identified regulatory partners of ATP7A and ATP7B that include the adaptor protein-1 complex. Upon knocking out pan-AP-1, sorting of both Cu-ATPases is disrupted. ATP7A loses its trafficking polarity and localizes on both apical and basolateral surfaces in high copper. By contrast, ATP7B loses TGN retention but retained its trafficking polarity to the apical domain, which became copper independent. Using isoform-specific knockouts, we found that the AP-1A complex provides directionality and TGN retention for both Cu-ATPases, whereas the AP-1B complex governs copper-independent trafficking of ATP7B solely. Trafficking phenotypes of Wilson disease-causing ATP7B mutants that disrupts putative ATP7B-AP1 interaction further substantiates the role of AP-1 in apical sorting of ATP7B.

312

项与金属代谢病相关的新闻（医药）

2024-04-03

·BioSpace

Xeris Pharmaceuticals Announces Inducement Grants Under NASDAQ Listing Rule 5635(c)(4) - April 03, 2024

CHICAGO--(BUSINESS WIRE)-- Xeris Biopharma Holdings, Inc. (Nasdaq: XERS), a growth-oriented biopharmaceutical company committed to improving patient lives by developing and commercializing innovative products across a range of therapies, today announced that on April 1, 2024, the Compensation Committee of Xeris’ Board of Directors granted restricted stock units for an aggregate of 103,000 shares of its common stock to 18 new employee(s) under Xeris’ Inducement Equity Plan. Xeris’ Inducement Equity Plan is used exclusively for the grant of equity awards to individuals who were not previously employed by Xeris or one of its subsidiaries as an inducement material to such individual's entering into employment with Xeris or one of its subsidiaries, pursuant to Rule 5635(c)(4) of the NASDAQ Listing Rules. The restricted stock units will vest over a period of three years in equal annual installments, and are subject to the employees’ continued employment with Xeris or one of its subsidiaries. All equity awards are subject to the terms and conditions of Xeris’ Inducement Equity Plan and forms of award agreements covering the grants. About Xeris Xeris (Nasdaq: XERS) is a growth-oriented biopharmaceutical company committed to improving patient lives by developing and commercializing innovative products across a range of therapies. Xeris has three commercially available products; Gvoke®, a ready-to-use liquid glucagon for the treatment of severe hypoglycemia, Keveyis®, a proven therapy for primary periodic paralysis, and Recorlev® for the treatment of endogenous Cushing’s syndrome. Xeris also has a robust pipeline of development programs to extend the current marketed products into important new indications and uses and bring new products forward using its proprietary formulation technology platforms, XeriSol™ and XeriJect®, supporting long-term product development and commercial success. Xeris Biopharma Holdings is headquartered in Chicago, IL. For more information, visit , or follow us on X, LinkedIn, or Instagram. View source version on businesswire.com: Contacts Investor Contact Allison Wey Senior Vice President, Investor Relations and Corporate Communications awey@xerispharma.com Source: Xeris Biopharma Holdings, Inc. View this news release online at:

2024-04-02

·GlobeNewswire-Health Care

Telomir Pharmaceuticals’ Anti-Aging Expert and NYT Bestselling Author Michael F. Roizen, MD, to Present Promising Pre-Clinical Data for Age-Reversal Drug Telomir-1 at the National Press Club in Washington, DC, on April 15, 2024

Telomir’s pre-clinical studies demonstrate its lead development product, Telomir-1, elongates telomere caps on human normal embryonic fibroblast cells by stimulating the enzyme telomerase New York Times article supports belief that telomere shortening reduces longevity BALTIMORE, April 02, 2024 (GLOBE NEWSWIRE) -- Telomir Pharmaceuticals, Inc. (Nasdaq:TELO) (“Telomir” or the “Company”), a pre-clinical-stage pharmaceutical company focused on the development and commercialization of Telomir-1 as the first novel small molecule to lengthen the DNA’s protective telomere caps in order to potentially address age-related conditions, today announced that anti-aging expert and New York Times bestselling author Michael F. Roizen, MD, will present promising pre-clinical research on Telomir-1 at the National Press Club in Washington, DC, on April 15, 2024. Dr. Roizen serves as Telomir’s special advisor on age reversal. Telomir’s pre-clinical research supports its belief that enlongating telomeres, which are repetitive DNA sequences at the end of chromosomes that protect chromosomes from becoming frayed or tangled, could stimulate stem cell renewal and provide a treatment for age-related conditions like osteoarthritis. The search for treatments to combat the diseases of aging has increasingly become the focus of many medical professionals and, more recently, society in general. In a March 20, 2024, article titled “Why Do We Age?”, The New York Times reported, “as we age, telomere shortening becomes a problem, particularly in stem cells, which the body uses to replenish skin, blood and other tissue.” Telomir believes that its lead development product, Telomir-1, may elongate telomeres which could reverse age-related conditions. “Our event at the National Press Club will provide us with an opportunity to share Telomir-1’s potential with the public on a national stage,” stated Chris Chapman, MD, co-founder, chairman, chief executive officer and president of Telomir Pharmaceuticals. “As part of the event, we will showcase our pre-clinical data which we believe demonstrates that Telomir-1 may potentially treat age-related conditions and prolong human life.” “Having recently shared our pre-clinical results with our peers at several longevity conferences, we believe that the opportunity to share our data with the public at this juncture is very important,” said Dr. Roizen. “Telomir-1’s results thus far have been extremely encouraging and, by presenting the data at this event, we are confident that the findings will be of interest to the entire longevity community. As Telomir continues to uncover new and promising data through our pre-clinical work, we remain committed to sharing our data publicly. I believe that the work that Telomir is undertaking to develop and commercialize Telomir-1 may finally provide an answer to the fight against diseases of aging.” To be added to the Telomir Pharmaceuticals email distribution list, please email telomir@kcsa.com with TELO in the subject line. About Telomir Pharmaceuticals, Inc. Telomir Pharmaceuticals, Inc. (Nasdaq:TELO) is a pre-clinical-stage pharmaceutical company focused on the development and commercialization of Telomir-1 as the first novel small molecule to lengthen the DNA’s protective telomere caps in order to potentially reverse age-related conditions. Telomeres are the protective end caps of a chromosome made up of DNA sequences and proteins. As humans age, telomeres shorten, with metal reactivity accelerating the process, which presents us with an increased chance of contracting a number of degenerative and age-related diseases. Telomir’s goal is to develop and commercialize Telomir-1 (which is proposed to be dosed orally) for hemochromatosis (iron overload) and ultimately post-chemotherapy recovery and a broader range of other age-related inflammatory conditions such as osteoarthritis. The Nobel Assembly at Karolinska Institute (Sweden) awarded the Nobel Prize in Physiology or Medicine in 2009 for the discovery of how chromosones are protected by telomeres and the enzyme telomerase. Cautionary Note Regarding Forward-Looking Statements This press release and the statements of the Company’s management related thereto contains “forward-looking statements,” which are statements other than historical facts made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding Telomir’s technologies potential in treating or reversing age-related conditions and Telomir-1’s ability to result in an individual’s ability to repair oneself by using Telomir-1. Any forward-looking statements in this press release are based on Telomir's current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties (many of which are beyond the Company’s control) that could cause actual results (including the anticipated benefits of the pre-clinical data and the Company’s presentation at the National Press Club as discussed herein) to differ materially and adversely from those set forth in or implied by such forward-looking statements. These and other risks concerning Telomir's programs and operations are described in additional detail in its Annual Report on Form 10-K for the year ended December 31, 2023, and other SEC filings, which are on file with the SEC at www.sec.gov and the Company’s website at https://ir.telomirpharma.com. Telomir explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. For further information, please contact: KCSA Strategic Communications Phil Carlsontelomir@kcsa.com Telomir Pharmaceuticalsinfo@Telomirpharma.com(813) 864-2558

2024-04-02

·BioSpace

Xeris Biopharma to Participate in the 23rd Annual Needham Virtual Healthcare Conference

CHICAGO--(BUSINESS WIRE)-- Xeris Biopharma Holdings, Inc. (Nasdaq: XERS), a growth-oriented biopharmaceutical company committed to improving patients’ lives by developing and commercializing innovative products across a range of therapies, today announced that Paul R. Edick, Xeris’ Chairman and CEO, will present an overview of the Company at the 23rd Annual Needham Virtual Healthcare Conference on Tuesday, April 9, 2024 at 11:00 am Eastern Time. To access the presentation, please visit “Events” on the investor relations page of the Company’s website at . About Xeris Xeris (Nasdaq: XERS) is a growth-oriented biopharmaceutical company committed to improving patients’ lives by developing and commercializing innovative products across a range of therapies. Xeris has three commercially available products; Gvoke®, a ready-to-use liquid glucagon for the treatment of severe hypoglycemia, Keveyis®, a proven therapy for primary periodic paralysis, and Recorlev® for the treatment of endogenous Cushing’s syndrome. Xeris also has a robust pipeline of development programs to extend the current marketed products into important new indications and uses and bring new products forward using its proprietary formulation technology platforms, XeriSol™ and XeriJect®, supporting long-term product development and commercial success. Xeris Biopharma Holdings is headquartered in Chicago, IL. For more information, visit , or follow us on X, LinkedIn, or Instagram. View source version on businesswire.com: Contacts Investor Contact Allison Wey Senior Vice President, Investor Relations and Corporate Communications awey@xerispharma.com Source: Xeris Biopharma Holdings, Inc. View this news release online at: