Updates on new therapies in development for rare liver diseases

(European Association for the Study of the Liver) Long-term data with sebelipase alfa for lysosomal acid lipase deficiency and preliminary data for investigational RNAi therapeutics for acute intermittent porphyria and alpha-1 antitrypsin deficiency add continued hope for the future management of metabolic and rare liver diseases.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news

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Publication date: Available online 15 October 2019Source: Stem Cell ResearchAuthor(s): Candice Ashmore-Harris, Samuel JI Blackford, Benjamin Grimsdell, Ewelina Kurtys, Marlies C Glatz, Tamir S Rashid, Gilbert O FruhwirthAbstractPrimary hepatocyte transplantation (HTx) is a safe cell therapy for patients with liver disease, but wider application is circumvented by poor cell engraftment due to limitations in hepatocyte quality and transplantation strategies. Hepatocyte-like cells (HLCs) derived from human induced pluripotent stem cells (hiPSC) are considered a promising alternative but also require optimisation of transplant...
Source: Stem Cell Research - Category: Stem Cells Source Type: research
This article explores different microbiota modulation strategies for the treatment of NAFLD-NASH.Alimentary Pharmacology &Therapeutics
Source: Medscape Today Headlines - Category: Consumer Health News Tags: Gastroenterology Journal Article Source Type: news
Authors: De Pace V, Morelli MC, Ravaioli M, Maggi F, Galli S, Vero V, Re MC, Cescon M, Pistello M Abstract Safety, efficacy, and predictor factors of sustained-virological-response after 24 weeks of new directacting antivirals were evaluated in hepatitis C virus patients with different stages of hepatic disease. 260 patients, median age 60 years, of whom 48.1% cirrhotics, 17.7% liver transplant recipients, and 45.7% naïve were treated with Sofosbuvir+Ribavirine, Sofosbuvir+Simeprevir±Ribavirine, Sofosbuvir+Daclatasvir±Ribavirine, Sofosbuvir+Ledispavir±Ribavirine, Ombitasvir/Paritaprevir/Ri...
Source: New Microbiologica - Category: Microbiology Tags: New Microbiol Source Type: research
Objectives The goals of this study were to compare the efficacy of the new manganese-based magnetic resonance imaging (MRI) contrast agent Mn-PyC3A to the commercial gadolinium-based agents Gd-DOTA and to Gd-EOB-DTPA to detect tumors in murine models of breast cancer and metastatic liver disease, respectively, and to quantify the fractional excretion and elimination of Mn-PyC3A in rats. Methods T1-weighted contrast-enhanced MRI with 0.1 mmol/kg Mn-PyC3A was compared with 0.1 mmol/kg Gd-DOTA in a breast cancer mouse model (n = 8) and to 0.025 mmol/kg Gd-EOB-DTPA in a liver metastasis mouse model (n = 6). The fractional...
Source: Investigative Radiology - Category: Radiology Tags: Original Articles Source Type: research
Authors: Avinash M, Renjith KR, Shetty AP, Sharma V, Kanna RM, Rajasekaran S Abstract Study design: Prospective study. Purpose: Last decade has witnessed the emergence of unplanned readmissions as a tool to gauge health-care quality and hospital performance. Previous studies were limited by their retrospective designs based on data-base information and short-term 30-day follow-up intervals. We analyzed incidence and causes for unplanned readmissions following spine surgery at 90-d interval and its difference at 30-d, 31-60-d and 61-90-d intervals, Total Bed-Days Lost (TBL) and economic impact of readmissions an...
Source: Asian Spine Journal - Category: Orthopaedics Tags: Asian Spine J Source Type: research
Source: BMJ Comments - Category: General Medicine Source Type: forums
ConclusionBBD could prevent and cure hepatic encephalopathy derived from both chronic and acute liver diseases. Here, we found that except for the direct improvement of blood and brain ammonia level, inflammation is likely an important target of BBD to treat hepatic encephalopathy. The anti-inflammatory role of BBD may lie in its regulation of the TLR4/MyD88/NF-κ B pathways.Graphical abstractBBD can prevent and cure hepatic encephalopathy derived from both chronic and acute liver diseases. BBD can reduce hyperammonemia as well as the systematic and neurological inflammation. Inflammation is likely an important target...
Source: Journal of Ethnopharmacology - Category: Drugs & Pharmacology Source Type: research
Conditions:   NAFLD;   Bariatric Surgery Candidate Intervention:   Procedure: Bariatric Surgery Sponsor:   Assiut University Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Authors: Villano G, Verardo A, Martini A, Brocco S, Pesce P, Novo E, Parola M, Sacerdoti D, Di Pascoli M, Fedrigo M, Castellani C, Angelini A, Pontisso P, Bolognesi M Abstract INTRODUCTION AND OBJECTIVES: SerpinB3 is a cysteine protease inhibitor involved in several biological activities. It is progressively expressed in chronic liver disease, but not in normal liver. The role in vascular reactivity of this serpin, belonging to the same family of Angiotensin II, is still unknown. Our aim was to evaluate the in vivo and in vitro effects of SerpinB3 on systemic and splanchnic hemodynamics. MATERIAL AND METHODS: D...
Source: Annals of Hepatology - Category: Gastroenterology Tags: Ann Hepatol Source Type: research
Authors: Barcelos STA, Dall'Oglio VM, de Araújo A, Cerski CTS, Álvares-da-Silva MR Abstract Hepatic sinusoidal obstruction syndrome (HSOS) is a hepatic vascular disease histologically characterized by edema, necrosis, detachment of endothelial cells in small sinusoidal hepatic and interlobular veins and intrahepatic congestion, which leads to portal hypertension and liver dysfunction. In the Western world, most HSOS cases are associated with myeloablative pretreatment in a hematopoietic stem cell transplantation setting. Here we report a case of a 54 years old female patient, otherwise healthy, with n...
Source: Annals of Hepatology - Category: Gastroenterology Tags: Ann Hepatol Source Type: research
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