Chronic Kidney Disease (CKD) affects around 10% of the adult population and is associated with an increased risk of heart attack, stroke and end stage kidney disease (ESKD). This study aims to better predict who is most likely to progress to ESKD using previously identified risk factors and novel biomarkers in blood and urine samples, along with kidney biopsy tissue. Resources can then be directed to those most at risk of disease progression and other associated conditions such as heart attack and stroke, while those at lower risk can be offered less frequent monitoring.

开始日期2016-07-27

申办/合作机构

The University of Nottingham

[+7]

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项与 UCB Pharma Ltd. 相关的文献（医药）

2024-06-01·Pediatric Neurology

Retrospective Multicenter Cohort Study on Safety and Electroencephalographic Response to Lacosamide for Neonatal Seizures

Article

作者: Elmoufti, Sami ; Kaur, Moninder ; Maltenfort, Mitchell G ; Pajor, Nathan M ; McDonald, Jill ; Utidjian, Levon ; Slaughter, Laurel ; Jain, Badal G ; Abend, Nicholas S ; Guerriero, Rejean M ; Foskett, Nadia ; Shellhaas, Renée A ; Dickinson, Kimberley ; Rao, Suchitra ; Roebling, Robert ; Forrest, Christopher B

BACKGROUNDThere is growing evidence supporting the safety and effectiveness of lacosamide in older children. However, minimal data are available for neonates. We aimed to determine the incidence of adverse events associated with lacosamide use and explore the electroencephalographic seizure response to lacosamide in neonates.METHODSA retrospective cohort study was conducted using data from seven pediatric hospitals from January 2009 to February 2020. For safety outcomes, neonates were followed for ≤30 days from index date. Electroencephalographic response of lacosamide was evaluated based on electroencephalographic reports for ≤3 days.RESULTSAmong 47 neonates, 98% received the first lacosamide dose in the intensive care units. During the median follow-up of 12 days, 19% of neonates died, and the crude incidence rate per 1000 patient-days (95% confidence interval) of the adverse events by diagnostic categories ranged from 2.8 (0.3, 10.2) for blood or lymphatic system disorders and nervous system disorders to 10.5 (4.2, 21.6) for cardiac disorders. Electroencephalographic seizures were observed in 31 of 34 patients with available electroencephalographic data on the index date. There was seizure improvement in 29% of neonates on day 1 and also in 29% of neonates on day 2. On day 3, there was no change in 50% of neonates and unknown change in 50% of neonates.CONCLUSIONSThe results are reassuring regarding the safety of lacosamide in neonates. Although some neonates had fewer seizures after lacosamide administration, the lack of a comparator arm and reliance on qualitative statements in electroencephalographic reports limit the preliminary efficacy results.

2024-05-01·Neurological Sciences

Using an expanded algorithm to estimate prevalence of amyotrophic lateral sclerosis in U.S. and UK

Article

作者: White, Richard ; Rudnik, Jan ; Vanderkelen, Mark ; Abbasi, Ali ; Fryk, Henrik ; Scowcroft, Anna ; Bonar, Kerina

BACKGROUNDThere is an increasing need to better understand the burden of amyotrophic lateral sclerosis (ALS) using real-world data (RWD). However, identifying ALS cases using RWD presents several challenges due to the rarity of ALS and the differences in database coding systems.METHODSMarketScan claims, and the UK Clinical Practice Research Datalink (CPRD) databases were searched for diagnosis codes of ALS or MND, the only drugs approved for treating ALS (riluzole and edaravone) and clinical visits with 12-month enrolment prior to 1 January 2011. The main algorithm required ≥ 1 ALS diagnosis code together with prescriptions or clinical visits. We expanded the existing algorithm to identify unspecific (possible) ALS group that had codes for motor neuron disease (MND) and the ALS drugs. The study period was from 1 January 2011 until 31 December 2020.RESULTSWe identified 16,246 patients with ≥ 1 ALS code in Marketscan (denominator n = 85,279,619), yet only 184 were found in the UK CPRD (denominator n = 21,318,589). Using the main algorithm 9,433 ALS patients were included in MarketScan, with a prevalence ranged between 4.5 per 100,000 in 2019 and 6.2 in 2015. In MarketScan, 3,658 (4.3 per 100,000) had ≥ 1 MND code and the ALS drug codes (possible cases). In CPRD, 47.9% of 2,785 patients with ≥ 1 MND code had a prescription for riluzole (6.3 per 100,000), regarded as possible ALS cases.CONCLUSIONSThe expanded algorithm enabled the identification of a large population with ALS, or possible ALS, and the estimation of ALS prevalence in MarketScan and CPRD.

2023-11-10·Oncogene

S100PBP is regulated by mutated KRAS and plays a tumour suppressor role in pancreatic cancer

Article

作者: Srivastava, K ; Jach, D ; Lines, K E ; Crnogorac-Jurcevic, T

AbstractWe have previously shown that expression of S100PBP, an S100P binding partner, gradually decreases during progression of pancreatic ductal adenocarcinomas (PDAC). Here, we show that loss of S100PBP leads to oncogenic transformation of pancreatic cells; after deregulation of S100PBP expression, both in silico and in vitro analyses highlighted alterations of genes known to modulate cytoskeleton, cell motility and survival. Overexpression of S100P reduced S100PBP expression, while co-immunoprecipitation indicated the interaction of S100P with S100PBP-p53-ubiquitin protein complex, likely causing S100PBP degradation. The doxycycline-induced KrasG12D activation resulted in decreased S100PBP levels, while low-dose treatment with HDAC inhibitor MS-275 rescued its expression in both human and mouse PDAC cell lines. This indicates KrasG12D as an upstream epigenetic regulator of S100PBP. Finally, analysis of TCGA PanCancer Atlas PDAC datasets demonstrated poor prognosis in patients with high S100P and low S100PBP expression, suggesting that S100PBP is a novel tumour suppressor gene with potential clinical utility.

项与 UCB Pharma Ltd. 相关的新闻（医药）

2024-10-29

·PRNewswire

Genascence Appoints Jeymi Tambiah as Chief Medical Officer

—Accomplished physician-scientist with more than 15 years' biopharmaceutical industry experience in medical affairs, clinical development, and drug development —Visionary leader skilled at leveraging scientific and clinical expertise to build innovative first-in-class therapeutic programs in areas of osteoarthritis, immunology, oncology, and neuroscience PALO ALTO, Calif., Oct. 29, 2024 /PRNewswire/ -- Genascence Corporation ("Genascence"), a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, today announced the appointment of Jeymi Tambiah, MBChB, FRCS, MS, FAPCR, MFPM(Dis) as its chief medical officer. Dr. Tambiah is an accomplished physician-scientist with more than 15 years' biopharmaceutical industry experience creating and leading strategies for medical affairs, clinical development, and drug development programs. He also brings deep experience in translational medicine, commercialization, and health outcomes research to Genascence. Continue Reading Jeymi Tambiah, Chief Medical Officer, Genascence "Jeymi is a visionary leader skilled at leveraging scientific and clinical expertise to build innovative first-in-class therapeutic programs in the areas of osteoarthritis, immunology, oncology, and neuroscience and I am excited to welcome him to Genascence as our chief medical offer," said Thomas Chalberg, Ph.D., founder and CEO of Genascence. "His experience includes all phases of drug development from bench through Phase 1-4 clinical studies and building elite high performance medical affairs teams. Jeymi's development and research expertise, particularly within the osteoarthritis research community, will be invaluable as we execute on our strategy to bring gene therapy to people suffering from prevalent musculoskeletal diseases." "Genascence is a company that is transforming how we treat debilitating musculoskeletal diseases like osteoarthritis by pioneering the development of gene therapy," said Dr. Tambiah. "I am thrilled to join this outstanding team under Tom's leadership and apply my knowledge and experience to advance our clinical program so that we can bring potentially curative therapies to patients in desperate need of better treatments." Before joining Genascence, Jeymi was senior vice president of clinical development and external innovation at Biosplice Therapeutics, a start-up biotech developing molecules modulating mRNA splicing in oncology, arthritis, and neurodegenerative diseases, where he was primarily responsible for clinical development, medical affairs, and external thoughtleader and scientific partnerships. Previously, Jeymi worked for UCB Pharma in various roles of increasing leadership within medical affairs, launching the TNF-inhibitor certolizumab pegol within the European Union, U.K., and U.S. across multiple immunology indications. Before his move into pharma, Jeymi trained and practiced in cardiothoracic surgery at Guys' and St. Thomas' Hospitals in London. Jeymi is very active in the osteoarthritis research community, with numerous research awards and publications, holding many committee appointments, and regularly presenting at international conferences. He is the North American representative for the Faculty of Pharmaceutical Medicine UK, and in this role is dedicated to supporting the education and mentoring of physicians in drug development. Jeymi earned his medical degree from the University of Manchester, UK, a doctorate degree in vascular biology from Imperial College London where he was a Wellcome Research Fellow, and a BSc (Hons) in medical science from the University of St. Andrews, UK. He is a fellow of the Royal College of Surgeons of England and Edinburgh, a member of Faculty of Pharmaceutical Physicians UK by Distinction, and a fellow of the Academy of Physicians in Clinical Research. About Genascence Corporation Genascence, a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, is developing life-changing treatments for highly prevalent conditions affecting millions of people. The company was founded in 2017 with technology licensed from three leading U.S. research institutions: Mayo Clinic, University of Florida, and NYU Langone Health. Headquartered in Palo Alto, California, Genascence's founders and leadership team have deep experience in the design, development, and manufacturing of successful gene therapies and biological medicines. For more information, please visit . SOURCE Genascence WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

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