PYC Progress Report by CEO Rohan Hockings and 2027 Commercialisation of PKD Drug 24 Nov 2023
COMMENTS
Polycystic kidney disease
Renal and extrarenal manifestations in polycystic kidney disease. a | Autosomal dominant polycystic kidney disease (ADPKD) is the most common form of polycystic kidney disease (PKD) and is mainly caused by mutations in PKD1 and PKD2, which encode polycystin 1 and polycystin 2, respectively.ADPKD is usually an adult-onset disease that is characterized by the formation of fluid-filled cysts in ...
Polycystic kidney disease
Polycystic kidney disease (PKD) is a genetic disorder whereby a large number of cysts develop primarily within the kidneys, but also in other organs. PKD can be autosomal dominant (one mutated ...
A new era in the science and care of kidney diseases
Well beyond kidney disease research, the major challenge will be to integrate genomics and/or transcriptomics discoveries into function and dysfunction at the organ and systemic level ...
Polycystic kidney disease
Michael Rehman. Stefan Somlo. Cystic kidneys are common causes of end-stage renal disease, both in children and in adults. Autosomal dominant polycystic kidney disease (ADPKD) and autosomal ...
Advances in Autosomal Dominant Polycystic Kidney Disease: A Clinical
Polycystic kidney disease (PKD) is a multiorgan disorder resulting in fluid-filled cyst formation in the kidneys and other systems. The replacement of kidney parenchyma with an ever-increasing volume of cysts eventually leads to kidney failure. Recently, increased understanding of the pathophysiology of PKD and genetic advances have led to new approaches of treatment targeting physiologic ...
Polycystic Kidney Disease
The two types of autosomal dominant polycystic kidney disease have similar pathological and physiological features, ... In vitro methods in renal research: In: Avner ED, Harmon WE, Niaudet P, eds ...
Experimental Models of Polycystic Kidney Disease: Applications and
Polycystic kidney diseases (PKDs) are genetic disorders characterized by the formation and expansion of numerous fluid-filled renal cysts, damaging normal parenchyma and often leading to kidney failure. Although PKDs comprise a broad range of different diseases, with substantial genetic and phenotypic heterogeneity, an association with primary ...
The Future of Polycystic Kidney Disease Research—As Seen By the 12
This article summarizes the contributions of the 12 Kaplan awardees and their vision for the future of PKD research. Keywords: genetic renal disease, polycystic kidney disease, genetics and development. In 2013, Jared J. Grantham, M.D. retired from the University of Kansas Medical Center (KUMC) after a long and distinguished career devoted to ...
Research priorities for autosomal dominant polycystic kidney disease: a
The Polycystic Kidney Disease Charity began a priority setting partnership with the James Lind Alliance (JLA) in the UK in 2019-2020 to identify areas of uncertainty in the ADPKD care pathway and allow patients, carers and healthcare professionals to rank the 10 most important questions for research. ... The use of both a paper and online ...
Review of the Use of Animal Models of Human Polycystic Kidney Disease
Autosomal dominant polycystic kidney disease, autosomal recessive polycystic kidney disease, and nephronophthisis are hereditary disorders with the occurrence of numerous cysts in both kidneys, often causing chronic and end-stage renal failure. Animal models have played an important role in recent advances in research not only on disease onset and progressive mechanisms but also on the ...
The pathophysiology and therapy of polycystic kidney disease
The intent of this special Call for Papers is to highlight research in polycystic kidney disease (PKD). PKD is a genetic disorder that causes many fluid-filled cysts to grow in human kidneys. The development of PKD cysts can change the shape of the kidneys, including making them much larger, and reduce kidney function, eventually leading to ...
International consensus statement on the diagnosis and ...
Autosomal dominant polycystic kidney disease (ADPKD) is the most common genetic disease in adults, with an estimated prevalence of 1 in 500-2,500 (refs 1,2,3,4).Cyst development starts early in ...
Research Suggests Avenues Toward Gene Therapies for Polycystic Kidney
Autosomal dominant polycystic kidney disease (ADPKD) is the most common potentially lethal genetic disease—about a half million people in the United States alone suffer from the condition There is no cure, but new research could open the door to new gene therapies for treating most cases of the disease. For several decades, researchers have known that mutations in the PKD1 gene, which ...
Autosomal Dominant Polycystic Kidney Disease
Polycystic kidney disease can be categorized into two forms based on inheritance patterns: autosomal dominant polycystic kidney disease and autosomal recessive polycystic kidney disease (ARPKD). ADPKD is more prevalent, affecting 1 in 400 to 1,000 people, while ARPKD occurs less frequently, with an estimated prevalence of 1 in 20,000 to 40,000 ...
A new drug candidate can shrink kidney cysts
A conversation with his brother, Sorin Fedeles, who studies polycystic kidney disease, led the pair to theorize that these compounds might also be good candidates for treating kidney cysts. At the time, research in ADPKD was beginning to suggest that kidney cyst cells also experience oxidative stress, due to an abnormal metabolism that ...
Oral delivery of nanomedicine for genetic kidney disease
USC/UKRO Kidney Research Center, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA. Search for other works by this author on: Oxford Academic. ... Chronic and genetic kidney diseases such as autosomal dominant polycystic kidney disease (ADPKD) have few therapeutic options, and clinical trials testing small ...
Polycystic kidney disease
Autosomal dominant polycystic kidney disease (ADPKD) is caused by pathogenic mutations in the genes that encode polycystin 1 (PC1) and PC2. In this article, the authors discuss findings from ...
Polycystic Kidney Disease Clinical Trials
A Study to Evaluate RGLS4326 to Treat Autosomal Dominant Polycystic Kidney Disease Rochester, MN. The primary objective of this study is to assess the dose response relationship between RGLS4326 and ADPKD biomarkers. Secondary objectives of this study are to characterize the pharmacokinetic (PK) properties of RGLS4326 in plasma and urine, and ...
Aneurysms of splenic artery in a patient with autosomal dominant
Kidney Int 88:17-27. Article PubMed PubMed Central Google Scholar Ruderman I, Menahem S (2014) Generalized aneurysmal disease in association with autosomal dominant polycystic disease. Clin Kidney J 7:416-417. Article PubMed PubMed Central Google Scholar Download references
Case report: Atypical polycystic kidney disease
Discussion. Atypical polycystic kidney disease (APKD) is a rare, benign condition that is indistinguishable from autosomal dominant polycystic kidney disease (ADPKD) both radiographically and histologically, but is currently recognized as a completely separate entity [2, 3]. It can be differentiated from ADPKD in several ways.
Coexisting presentation of two rare genetic variants of autosomal
A case of a man in his 60s with progressive chronic kidney disease, bilateral sensorineural hearing loss and multiple renal cysts, which suggests a potential synergic effect of both variants, which warrants further investigation. Alport syndrome and autosomal dominant polycystic kidney disease are monogenic causes of chronic kidney disease and end-stage kidney failure. We present a case of a ...
Association between Female Reproductive Factors and Risk of Dementia
Women have an over 50% greater risk of dementia than men, which is a main topic of much research. This review aims to investigate the impact of a woman's reproductive history on dementia risk. The consequences of stillbirth are long-term health and psychosocial problems for women. Because of the awareness of an endangered pregnancy, many parents experience deep anxiety and stress in ...
Chronic Kidney Disease Diagnosis and Management
Chronic kidney disease (CKD) affects between 8% and 16% of the population worldwide and is often underrecognized by patients and clinicians. 1-4 Defined by a glomerular filtration rate (GFR) of less than 60 mL/min/1.73 m 2, albuminuria of at least 30 mg per 24 hours, or markers of kidney damage (eg, hematuria or structural abnormalities such as polycystic or dysplastic kidneys) persisting ...
IMAGES
VIDEO
COMMENTS
Renal and extrarenal manifestations in polycystic kidney disease. a | Autosomal dominant polycystic kidney disease (ADPKD) is the most common form of polycystic kidney disease (PKD) and is mainly caused by mutations in PKD1 and PKD2, which encode polycystin 1 and polycystin 2, respectively.ADPKD is usually an adult-onset disease that is characterized by the formation of fluid-filled cysts in ...
Polycystic kidney disease (PKD) is a genetic disorder whereby a large number of cysts develop primarily within the kidneys, but also in other organs. PKD can be autosomal dominant (one mutated ...
Well beyond kidney disease research, the major challenge will be to integrate genomics and/or transcriptomics discoveries into function and dysfunction at the organ and systemic level ...
Michael Rehman. Stefan Somlo. Cystic kidneys are common causes of end-stage renal disease, both in children and in adults. Autosomal dominant polycystic kidney disease (ADPKD) and autosomal ...
Polycystic kidney disease (PKD) is a multiorgan disorder resulting in fluid-filled cyst formation in the kidneys and other systems. The replacement of kidney parenchyma with an ever-increasing volume of cysts eventually leads to kidney failure. Recently, increased understanding of the pathophysiology of PKD and genetic advances have led to new approaches of treatment targeting physiologic ...
The two types of autosomal dominant polycystic kidney disease have similar pathological and physiological features, ... In vitro methods in renal research: In: Avner ED, Harmon WE, Niaudet P, eds ...
Polycystic kidney diseases (PKDs) are genetic disorders characterized by the formation and expansion of numerous fluid-filled renal cysts, damaging normal parenchyma and often leading to kidney failure. Although PKDs comprise a broad range of different diseases, with substantial genetic and phenotypic heterogeneity, an association with primary ...
This article summarizes the contributions of the 12 Kaplan awardees and their vision for the future of PKD research. Keywords: genetic renal disease, polycystic kidney disease, genetics and development. In 2013, Jared J. Grantham, M.D. retired from the University of Kansas Medical Center (KUMC) after a long and distinguished career devoted to ...
The Polycystic Kidney Disease Charity began a priority setting partnership with the James Lind Alliance (JLA) in the UK in 2019-2020 to identify areas of uncertainty in the ADPKD care pathway and allow patients, carers and healthcare professionals to rank the 10 most important questions for research. ... The use of both a paper and online ...
Autosomal dominant polycystic kidney disease, autosomal recessive polycystic kidney disease, and nephronophthisis are hereditary disorders with the occurrence of numerous cysts in both kidneys, often causing chronic and end-stage renal failure. Animal models have played an important role in recent advances in research not only on disease onset and progressive mechanisms but also on the ...
The intent of this special Call for Papers is to highlight research in polycystic kidney disease (PKD). PKD is a genetic disorder that causes many fluid-filled cysts to grow in human kidneys. The development of PKD cysts can change the shape of the kidneys, including making them much larger, and reduce kidney function, eventually leading to ...
Autosomal dominant polycystic kidney disease (ADPKD) is the most common genetic disease in adults, with an estimated prevalence of 1 in 500-2,500 (refs 1,2,3,4).Cyst development starts early in ...
Autosomal dominant polycystic kidney disease (ADPKD) is the most common potentially lethal genetic disease—about a half million people in the United States alone suffer from the condition There is no cure, but new research could open the door to new gene therapies for treating most cases of the disease. For several decades, researchers have known that mutations in the PKD1 gene, which ...
Polycystic kidney disease can be categorized into two forms based on inheritance patterns: autosomal dominant polycystic kidney disease and autosomal recessive polycystic kidney disease (ARPKD). ADPKD is more prevalent, affecting 1 in 400 to 1,000 people, while ARPKD occurs less frequently, with an estimated prevalence of 1 in 20,000 to 40,000 ...
A conversation with his brother, Sorin Fedeles, who studies polycystic kidney disease, led the pair to theorize that these compounds might also be good candidates for treating kidney cysts. At the time, research in ADPKD was beginning to suggest that kidney cyst cells also experience oxidative stress, due to an abnormal metabolism that ...
USC/UKRO Kidney Research Center, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA. Search for other works by this author on: Oxford Academic. ... Chronic and genetic kidney diseases such as autosomal dominant polycystic kidney disease (ADPKD) have few therapeutic options, and clinical trials testing small ...
Autosomal dominant polycystic kidney disease (ADPKD) is caused by pathogenic mutations in the genes that encode polycystin 1 (PC1) and PC2. In this article, the authors discuss findings from ...
A Study to Evaluate RGLS4326 to Treat Autosomal Dominant Polycystic Kidney Disease Rochester, MN. The primary objective of this study is to assess the dose response relationship between RGLS4326 and ADPKD biomarkers. Secondary objectives of this study are to characterize the pharmacokinetic (PK) properties of RGLS4326 in plasma and urine, and ...
Kidney Int 88:17-27. Article PubMed PubMed Central Google Scholar Ruderman I, Menahem S (2014) Generalized aneurysmal disease in association with autosomal dominant polycystic disease. Clin Kidney J 7:416-417. Article PubMed PubMed Central Google Scholar Download references
Discussion. Atypical polycystic kidney disease (APKD) is a rare, benign condition that is indistinguishable from autosomal dominant polycystic kidney disease (ADPKD) both radiographically and histologically, but is currently recognized as a completely separate entity [2, 3]. It can be differentiated from ADPKD in several ways.
A case of a man in his 60s with progressive chronic kidney disease, bilateral sensorineural hearing loss and multiple renal cysts, which suggests a potential synergic effect of both variants, which warrants further investigation. Alport syndrome and autosomal dominant polycystic kidney disease are monogenic causes of chronic kidney disease and end-stage kidney failure. We present a case of a ...
Women have an over 50% greater risk of dementia than men, which is a main topic of much research. This review aims to investigate the impact of a woman's reproductive history on dementia risk. The consequences of stillbirth are long-term health and psychosocial problems for women. Because of the awareness of an endangered pregnancy, many parents experience deep anxiety and stress in ...
Chronic kidney disease (CKD) affects between 8% and 16% of the population worldwide and is often underrecognized by patients and clinicians. 1-4 Defined by a glomerular filtration rate (GFR) of less than 60 mL/min/1.73 m 2, albuminuria of at least 30 mg per 24 hours, or markers of kidney damage (eg, hematuria or structural abnormalities such as polycystic or dysplastic kidneys) persisting ...