Nicholas J.A. Webb
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View article: Oral iptacopan therapy in patients with C3 glomerulopathy: a randomised, double-blind, parallel group, multicentre, placebo-controlled, phase 3 study
Oral iptacopan therapy in patients with C3 glomerulopathy: a randomised, double-blind, parallel group, multicentre, placebo-controlled, phase 3 study Open
Novartis Pharma.
View article: Polygenic Risk Scores and HLA Class II Variants are Biomarkers of Corticosteroid Response in Childhood Nephrotic Syndrome
Polygenic Risk Scores and HLA Class II Variants are Biomarkers of Corticosteroid Response in Childhood Nephrotic Syndrome Open
Introduction Nephrotic syndrome (NS), a common glomerular disease in children, is classified based on response to corticosteroid therapy as either steroid-sensitive nephrotic syndrome (SSNS), or steroid-resistant nephrotic syndrome (SRNS).…
View article: Quantifying association of early proteinuria and estimated glomerular filtration rate changes with long-term kidney failure in C3 glomerulopathy and immune-complex membranoproliferative glomerulonephritis using the United Kingdom RaDaR Registry
Quantifying association of early proteinuria and estimated glomerular filtration rate changes with long-term kidney failure in C3 glomerulopathy and immune-complex membranoproliferative glomerulonephritis using the United Kingdom RaDaR Registry Open
Overall, proteinuria a short time after diagnosis is strongly associated with long-term outcomes and a UPCR under 100 mg/mmol at one year is associated with a substantially lower kidney failure risk.
View article: The prevalence of immunoglobulin A nephropathy in the European Union and the impact of the COVID-19 pandemic: an estimation approach utilizing the kidney biopsy frequency
The prevalence of immunoglobulin A nephropathy in the European Union and the impact of the COVID-19 pandemic: an estimation approach utilizing the kidney biopsy frequency Open
Background Immunoglobulin A nephropathy (IgAN) is the most common type of primary glomerulonephritis, requiring a kidney biopsy for diagnosis. This study aimed to estimate the prevalence of primary IgAN within the European Union (EU) and i…
View article: Kidney Transplantation in Children and Adolescents With <scp>C3</scp> Glomerulopathy or Immune Complex Membranoproliferative Glomerulonephritis: An International Survey of Current Practice
Kidney Transplantation in Children and Adolescents With <span>C3</span> Glomerulopathy or Immune Complex Membranoproliferative Glomerulonephritis: An International Survey of Current Practice Open
Background Approximately 50% of patients with chronic kidney disease due to C3 glomerulopathy (C3G) or primary immune‐complex membranoproliferative glomerulonephritis (IC‐MPGN) will require dialysis and/or kidney transplantation (KTx) with…
View article: Factor B as a therapeutic target for the treatment of complement-mediated diseases
Factor B as a therapeutic target for the treatment of complement-mediated diseases Open
The complement system, consisting of three initiating pathways—classical, lectin and alternative, is an important part of innate immunity. Dysregulation of the complement system is implicated in the pathogenesis of several autoimmune and i…
View article: Iptacopan Reduces Proteinuria and Stabilizes Kidney Function in C3 Glomerulopathy
Iptacopan Reduces Proteinuria and Stabilizes Kidney Function in C3 Glomerulopathy Open
These data provide a clinical rationale for further evaluation of long-term treatment of C3G with iptacopan.
View article: #277 Update to the long-term safety and efficacy of iptacopan in C3G: 33-month extension study data from patients enrolled in a phase 2 study
#277 Update to the long-term safety and efficacy of iptacopan in C3G: 33-month extension study data from patients enrolled in a phase 2 study Open
Background and Aims Complement 3 glomerulopathy (C3G), due to dysregulation of the alternative complement pathway (AP), is an ultra-rare primary glomerulonephritis. Iptacopan (LNP023) is an oral, proximal complement inhibitor that specific…
View article: WCN24-1146 SAFETY AND EFFICACY OF IPTACOPAN IN ADOLESCENT PATIENTS WITH IDIOPATHIC (PRIMARY) IMMUNE-COMPLEX-MEDIATED MEMBRANOPROLIFERATIVE GLOMERULONEPHRITIS (IC-MPGN)
WCN24-1146 SAFETY AND EFFICACY OF IPTACOPAN IN ADOLESCENT PATIENTS WITH IDIOPATHIC (PRIMARY) IMMUNE-COMPLEX-MEDIATED MEMBRANOPROLIFERATIVE GLOMERULONEPHRITIS (IC-MPGN) Open
IC-MPGN is an ultra-rare, fast-progressing complement-mediated kidney disease characterized by immunoglobulin deposits in the kidneys, which may be idiopathic (primary) or secondary to chronic infections, autoimmune disorders, or monoclona…
View article: Quantifying association of early proteinuria and eGFR changes with long-term kidney failure hazard in C3G and IC-MPGN
Quantifying association of early proteinuria and eGFR changes with long-term kidney failure hazard in C3G and IC-MPGN Open
Background C3 glomerulopathy (C3G) and immune-complex membranoproliferative glomerulonephritis (IC-MPGN) are rare disorders that frequently result in kidney failure over the long-term. At present, there are no disease-specific treatments a…
View article: Detection, Monitoring, and Mitigation of Drug-Induced Nephrotoxicity: A Pragmatic Approach
Detection, Monitoring, and Mitigation of Drug-Induced Nephrotoxicity: A Pragmatic Approach Open
The kidneys play a pivotal role in elimination of most drugs; therefore, a comprehensive understanding of renal physiology and pathology is important for those involved in drug development. High filtration capacity and metabolic activity m…
View article: Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy
Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy Open
Iptacopan resulted in statistically significant and clinically important reductions in UPCR and normalization of serum C3 levels in the native cohort and reduced C3 deposit scores in the recurrent KT cohort with favorable safety and tolera…
View article: Evaluating the Acceptability, Swallowability, and Palatability of Film-Coated Mini-Tablet Formulation in Young Children: Results from an Open-Label, Single-Dose, Cross-Over Study
Evaluating the Acceptability, Swallowability, and Palatability of Film-Coated Mini-Tablet Formulation in Young Children: Results from an Open-Label, Single-Dose, Cross-Over Study Open
Mini-tablets are advantageous over liquid formulations in overcoming challenges related to stability, taste, and dosage. This open-label, single-dose, cross-over study investigated the acceptability and safety of drug-free, film-coated min…
View article: Alternative Complement Pathway Inhibition With Iptacopan for the Treatment of C3 Glomerulopathy-Study Design of the APPEAR-C3G Trial
Alternative Complement Pathway Inhibition With Iptacopan for the Treatment of C3 Glomerulopathy-Study Design of the APPEAR-C3G Trial Open
This study aims to demonstrate the clinical benefits of AP inhibition with iptacopan in C3G.
View article: The pediatric glucocorticoid toxicity index
The pediatric glucocorticoid toxicity index Open
To develop a Pediatric glucocorticoid toxicity index (pGTI), a standardized, weighted clinical outcome assessment that measures change in glucocorticoid (GC) toxicity over time. Fourteen physician experts from 7 subspecialties participated…
View article: Lower Leg Power and Grip Strength Are Associated With Increased Fall Injury Risk in Older Men: The Osteoporotic Fractures in Men Study
Lower Leg Power and Grip Strength Are Associated With Increased Fall Injury Risk in Older Men: The Osteoporotic Fractures in Men Study Open
Background Past research has not investigated both lower-extremity power and upper-extremity strength in the same fall injury study, particularly nonfracture fall injuries. Methods In the Osteoporotic Fractures in Men Study (baseline: N = …
View article: POS-038 ALTERNATIVE COMPLEMENT PATHWAY INHIBITION WITH IPTACOPAN TO ARREST DISEASE PROGRESSION IN C3 GLOMERULOPATHY (APPEAR-C3G): A PHASE 3 STUDY
POS-038 ALTERNATIVE COMPLEMENT PATHWAY INHIBITION WITH IPTACOPAN TO ARREST DISEASE PROGRESSION IN C3 GLOMERULOPATHY (APPEAR-C3G): A PHASE 3 STUDY Open
IntroductionComplement 3 glomerulopathy (C3G) is a rare kidney disease characterized by dysregulation of the alternative pathway (AP) of the complement system. Around 50% of patients progress to kidney failure within 10 years of diagnosis.…
View article: Expert guidance on the multidisciplinary management of cystinosis in adolescent and adult patients
Expert guidance on the multidisciplinary management of cystinosis in adolescent and adult patients Open
Cystinosis, a rare autosomal recessive lysosomal storage disorder, results in an abnormal accumulation of the amino acid cystine in multiple organs and tissues of the body. Renal symptoms typically develop in the first few months of life, …
View article: Evaluation of Daily Low-Dose Prednisolone During Upper Respiratory Tract Infection to Prevent Relapse in Children With Relapsing Steroid-Sensitive Nephrotic Syndrome
Evaluation of Daily Low-Dose Prednisolone During Upper Respiratory Tract Infection to Prevent Relapse in Children With Relapsing Steroid-Sensitive Nephrotic Syndrome Open
isrctn.org Identifier: ISRCTN10900733; EudraCT 2012-003476-39.
View article: FC 132SHORT COURSE DAILY LOW-DOSE PREDNISOLONE AT THE TIME OF UPPER RESPIRATORY TRACT INFECTION (URTI) IN NON-SELECTED CHILDREN WITH RELAPSING STEROID SENSITIVE NEPHROTIC SYNDROME DOES NOT PREVENT URTI-RELATED RELAPSE: THE PREDNOS 2 TRIAL
FC 132SHORT COURSE DAILY LOW-DOSE PREDNISOLONE AT THE TIME OF UPPER RESPIRATORY TRACT INFECTION (URTI) IN NON-SELECTED CHILDREN WITH RELAPSING STEROID SENSITIVE NEPHROTIC SYNDROME DOES NOT PREVENT URTI-RELATED RELAPSE: THE PREDNOS 2 TRIAL Open
Background and Aims At least 80% of children with steroid sensitive nephrotic syndrome (SSNS) have relapses and many are triggered by upper respiratory tract infections (URTIs). Previous small studies (4 studies, 232 patients in total), mo…
View article: POS-288 THE BURDEN OF HEART FAILURE IN PATIENTS WITH NON–DIALYSIS-DEPENDENT ANAEMIA OF CHRONIC KIDNEY DISEASE RECEIVING ERYTHROPOIESIS-STIMULATING AGENTS: A SYSTEMATIC LITERATURE REVIEW
POS-288 THE BURDEN OF HEART FAILURE IN PATIENTS WITH NON–DIALYSIS-DEPENDENT ANAEMIA OF CHRONIC KIDNEY DISEASE RECEIVING ERYTHROPOIESIS-STIMULATING AGENTS: A SYSTEMATIC LITERATURE REVIEW Open
Anaemia of chronic kidney disease (CKD) is associated with significant morbidity and increased mortality, particularly cardiovascular complications such as heart failure (HF). We performed a systematic literature review of the treatment op…
View article: Consultant-led triage of paediatric hospital referrals: a service evaluation
Consultant-led triage of paediatric hospital referrals: a service evaluation Open
Objective We established a paediatric demand management (PDM) service in our paediatric department in 2017. The aim of this consultant-delivered service is to manage referrals more efficiently by providing active triage of all referrals, d…