This gives me such hope and don't know why we have not gotten together like this before....
There are 90,000 people in the United States waiting for a kidney and all it takes is one stranger to begin a chain of events that has the potential to save an ever-growing number of lives.
Rick Ruzzamenti was the first link in the longest such chain so far -- known in this case as chain No. 124, as The New York Times first reported, a stranger who began a domino effect of generosity spanning across the country.
Click Here to Learn More About the National Kidney Registry
Click here for full story and video from ABC News!
Tuesday, February 21, 2012
Thursday, January 12, 2012
Identification of Novel Mutations in Chinese Hans with Autosomal Dominant Polycystic Kidney Disease
Identification of Novel Mutations in Chinese Hans with Autosomal Dominant Polycystic Kidney Disease
http://7thspace.com/headlines/402485/identification_of_novel_mutations_in_chinese_hans_with_autosomal_dominant_polycystic_kidney_disease.html
Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited renal disease with an incidence of 1 in 400 to 1000. The disease is genetically heterogeneous, with two genes identified: PKD1 (16p13.3) and PKD2 (4q21).
Molecular diagnosis of the disease in at-risk individuals is complicated due to the structural complexity of PKD1 gene and the high diversity of the mutations. This study is the first systematic ADPKD mutation analysis of both PKD1 and PKD2 genes in Chinese patients using denaturing high-performance liquid chromatography (DHPLC).
Methods: Both PKD1 and PKD2 genes were mutation screened in each proband from 65 families using DHPLC followed by DNA sequencing.
Novel variations found in the probands were checked in their family members available and 100 unrelated normal controls. Then the pathogenic potential of the variations of unknown significance was examined by evolutionary comparison, effects of amino acid substitutions on protein structure, and effects of splice site alterations using online mutation prediction resources.
Results: A total of 92 variations were identified, including 27 reported previously.
Definitely pathogenic mutations (ten frameshift, ten nonsense, two splicing defects and one duplication) were identified in 28 families, and probably pathogenic mutations were found in an additional six families, giving a total detection level of 52.3% (34/65). About 69% (20/29) of the mutations are first reported with a recurrent mutation rate of 31%.
Conclusions: Mutation study of PKD1 and PKD2 genes in Chinese Hans with ADPKD may contribute to a better understanding of the genetic diversity between different ethnic groups and enrich the mutation database.
Besides, evaluating the pathogenic potential of novel variations should also facilitate the clinical diagnosis and genetic counseling of the disease.
Author: Chaowen YuYuan YangLin ZouZhangxue HuJing LiYunqiang LiuYongxin MaMingyi MaDan SuSizhong Zhang
Credits/Source: BMC Medical Genetics 2011, 12:164
http://7thspace.com/headlines/402485/identification_of_novel_mutations_in_chinese_hans_with_autosomal_dominant_polycystic_kidney_disease.html
Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited renal disease with an incidence of 1 in 400 to 1000. The disease is genetically heterogeneous, with two genes identified: PKD1 (16p13.3) and PKD2 (4q21).
Molecular diagnosis of the disease in at-risk individuals is complicated due to the structural complexity of PKD1 gene and the high diversity of the mutations. This study is the first systematic ADPKD mutation analysis of both PKD1 and PKD2 genes in Chinese patients using denaturing high-performance liquid chromatography (DHPLC).
Methods: Both PKD1 and PKD2 genes were mutation screened in each proband from 65 families using DHPLC followed by DNA sequencing.
Novel variations found in the probands were checked in their family members available and 100 unrelated normal controls. Then the pathogenic potential of the variations of unknown significance was examined by evolutionary comparison, effects of amino acid substitutions on protein structure, and effects of splice site alterations using online mutation prediction resources.
Results: A total of 92 variations were identified, including 27 reported previously.
Definitely pathogenic mutations (ten frameshift, ten nonsense, two splicing defects and one duplication) were identified in 28 families, and probably pathogenic mutations were found in an additional six families, giving a total detection level of 52.3% (34/65). About 69% (20/29) of the mutations are first reported with a recurrent mutation rate of 31%.
Conclusions: Mutation study of PKD1 and PKD2 genes in Chinese Hans with ADPKD may contribute to a better understanding of the genetic diversity between different ethnic groups and enrich the mutation database.
Besides, evaluating the pathogenic potential of novel variations should also facilitate the clinical diagnosis and genetic counseling of the disease.
Author: Chaowen YuYuan YangLin ZouZhangxue HuJing LiYunqiang LiuYongxin MaMingyi MaDan SuSizhong Zhang
Credits/Source: BMC Medical Genetics 2011, 12:164
Friday, December 09, 2011
Relaxin improves poor blood flow and kidney function in a rat model of PKD
Published on December 7, 2011 at 8:02 AM
Research presented at ASCB annual meeting
After a four-week course of the vasodilator hormone relaxin, kidney function and blood flow immediately improved in lab rats genetically altered to model polycystic kidney disease (PKD), a life-threatening genetic disorder, according to research presented on Dec. 6 at the American Society for Cell Biology Annual Meeting in Denver.
In addition to widening the blood vessels, relaxin lowered the collagen scores of the PKD rats, indicating that the drug had slowed scar formation or helped dissolve the old fibroid tissue that characterizes the kidneys of animals and humans with the disease, according to Heather Ward, Ph.D., and Angela Wandinger-Ness, Ph.D., of the University of New Mexico and collaborators.
PKD is a life-threatening genetic disorder that affects 600,000 Americans, according to the National Institute of Diabetes, Digestive, and Kidney Diseases (NIDDK). About 50% of individuals diagnosed with PKD develop end-stage renal disease by age 60.
The researchers also noted that in rats, relaxin reduced the size of the large fluid-filled cysts that gradually encroach on kidney function in human PKD patients.
PKD was the first disease to be recognized as a ciliopathy, a disorder characterized by defects in primary cilia, tiny hair-like structures that protrude from virtually every cell in the human body.
In the search of effective treatments, most PKD researchers have concentrated on halting or reversing PKD's characteristic cyst formation.
Read More: http://www.news-medical.net/news/20111207/Relaxin-improves-poor-blood-flow-and-kidney-function-in-a-rat-model-of-PKD.aspx?page=2
Research presented at ASCB annual meeting
After a four-week course of the vasodilator hormone relaxin, kidney function and blood flow immediately improved in lab rats genetically altered to model polycystic kidney disease (PKD), a life-threatening genetic disorder, according to research presented on Dec. 6 at the American Society for Cell Biology Annual Meeting in Denver.
In addition to widening the blood vessels, relaxin lowered the collagen scores of the PKD rats, indicating that the drug had slowed scar formation or helped dissolve the old fibroid tissue that characterizes the kidneys of animals and humans with the disease, according to Heather Ward, Ph.D., and Angela Wandinger-Ness, Ph.D., of the University of New Mexico and collaborators.
PKD is a life-threatening genetic disorder that affects 600,000 Americans, according to the National Institute of Diabetes, Digestive, and Kidney Diseases (NIDDK). About 50% of individuals diagnosed with PKD develop end-stage renal disease by age 60.
The researchers also noted that in rats, relaxin reduced the size of the large fluid-filled cysts that gradually encroach on kidney function in human PKD patients.
PKD was the first disease to be recognized as a ciliopathy, a disorder characterized by defects in primary cilia, tiny hair-like structures that protrude from virtually every cell in the human body.
In the search of effective treatments, most PKD researchers have concentrated on halting or reversing PKD's characteristic cyst formation.
Read More: http://www.news-medical.net/news/20111207/Relaxin-improves-poor-blood-flow-and-kidney-function-in-a-rat-model-of-PKD.aspx?page=2
Saturday, November 26, 2011
Just the facts about Polycystic Kidney Disease
Just the facts about Polycystic Kidney Disease (PKD) - The most common life-threatening genetic disease in the world. It affects 600,000 Amercians and over 12.5 million people worldwide. Polycystic Kidney Disease (PKD) is more common than muscular distrophy, cystic fibrosis, down's syndrome, sickle cell anemia and hemophilia; combined. There is hope - Learn more at http://pkdcure.org/
Monday, November 21, 2011
Facts About Kidney Disease: Causes, Symptoms, and Diet
(2011-11-16) The kidneys are required to help maintain fluid balance, but in the case of kidney damage fluids may need to be restricted. This article discusses why fluid assessment is an essential part of a kidney
Your kidneys play very important roles in body functions. Hence it is important to maintain a healthy kidney diet. Kidneys filter the blood and get rid of waste products of your body like uric acid from the breakdown of DNA, and urea from the metabolism of protein. At the same time they balance the levels of electrolyte in the body such as potassium and sodium, and also control blood pressure. Kidneys also supply the hormone called erythropoietin, which helps stimulate the bone marrow to produce red blood cells.
Sometimes no matter how careful you are with your kidneys, there are still chances that you will develop kidney problems. Here are some of the causes of kidney disease:
* High blood pressure – also known as hypertension, if uncontrolled, the kidneys will be damaged over time.
* Diabetes –type 1 and 2 diabetes can lead to a condition called diabetic nephropathy which is the major cause of kidney disease.
* Hereditary –if you have a family member who has a history of kidney disease then you are susceptible with the same disease. One example of this condition is the polycystic kidney disease wherein the two kidneys develop cysts.
* Glomerulonephritis –the inflammation or damage of the kidney’s filtration system. This condition if not treated immediately can cause kidney failure.
* Long term medications –some medications can be toxic to the tissues of the kidney which can lead to kidney failure over time.
* Other infections –include certain cancers, sickle cell disease, amyloidosis, heroine abuse, and HIV infection
If one of these conditions is present in your body then you are at a high risk of developing kidney disease. Your kidneys should be regularly monitored, but some changes can be made to lessen your chance of developing kidney failure treatment. It can include a change in lifestyle and diet. If you already have kidney disease, then you need to have constant medication, or a better natural option would be following a strict kidney diet. But how would you know if you already have a kidney disease? Here are the following symptoms you can take a look at:
* Swollen eyes, feet and hands (this condition is commonly known as edema)
* Loss of appetite
* Constant tiredness
* High blood pressure
* Constant feeling of thirst
* Shortness of breath
* Bad breath
* Weight loss
* Nausea and vomiting
* Constant itchiness of the skin
* Muscle cramps
* Tea-colored or cloudy urine
* A yellowish-brown trace on the skin
There are two types of kidney failure, chronic kidney failure or acute kidney failure. Both types can be life-threatening. In both circumstances, the kidneys stop functioning normally and cannot filter wastes or too much water from the blood. Due to this condition, toxic materials start to build up which can cause several complications that can affect different body systems. People who suffer from chronic kidney failure will need kidney transplant or dialysis to be able to survive. If you have kidney failure, or you know someone who has this disease, a strict kidney diet must be followed.
* Drink at least 8-10 glasses of water every day. The main function of your kidneys is to filter toxins from the blood and drain them off through urine. Without adequate amount of water, your kidneys can’t function very well. Generally, most chronic kidney disease and kidney stones are caused by inadequate water intake. You can also eat fruits and vegetables which contain 90 percent of water like cucumber, tomatoes, watermelon and zucchini.
* Eat more vegetables than meat. Too much protein intake can stress your kidneys.
* Avoid foods that have high levels of cholesterol. Eat nondairy products and lean meats instead. Also include whole grains in your kidney diet.
* Avoid goods that are heavily sweetened.
* Quit smoking. Smoking can increase blood pressure which is one of the major causes of kidney disease.
When planning a kidney diet it is still best to consult your physician or a dietician. The right kidney diet plan for you will also depend on the condition that you have. The key to a healthy kidney diet is dedication and the right people to back you up.
About The Publisher:
Kidney is one of the most important organs in our body, And having the right solution for the problem in one’s kidney is very hard to find due to different ideas about it. Kidney Diet have many ways in treating them and we just need to know and to perform them correctly. For more information visit my site at http://mykidneydiet.com/
About Us: http://mykidneydiet.com/
Contact Info: +6519281414
http://www.addpr.com/articles/health_fitness/95704.html
Your kidneys play very important roles in body functions. Hence it is important to maintain a healthy kidney diet. Kidneys filter the blood and get rid of waste products of your body like uric acid from the breakdown of DNA, and urea from the metabolism of protein. At the same time they balance the levels of electrolyte in the body such as potassium and sodium, and also control blood pressure. Kidneys also supply the hormone called erythropoietin, which helps stimulate the bone marrow to produce red blood cells.
Sometimes no matter how careful you are with your kidneys, there are still chances that you will develop kidney problems. Here are some of the causes of kidney disease:
* High blood pressure – also known as hypertension, if uncontrolled, the kidneys will be damaged over time.
* Diabetes –type 1 and 2 diabetes can lead to a condition called diabetic nephropathy which is the major cause of kidney disease.
* Hereditary –if you have a family member who has a history of kidney disease then you are susceptible with the same disease. One example of this condition is the polycystic kidney disease wherein the two kidneys develop cysts.
* Glomerulonephritis –the inflammation or damage of the kidney’s filtration system. This condition if not treated immediately can cause kidney failure.
* Long term medications –some medications can be toxic to the tissues of the kidney which can lead to kidney failure over time.
* Other infections –include certain cancers, sickle cell disease, amyloidosis, heroine abuse, and HIV infection
If one of these conditions is present in your body then you are at a high risk of developing kidney disease. Your kidneys should be regularly monitored, but some changes can be made to lessen your chance of developing kidney failure treatment. It can include a change in lifestyle and diet. If you already have kidney disease, then you need to have constant medication, or a better natural option would be following a strict kidney diet. But how would you know if you already have a kidney disease? Here are the following symptoms you can take a look at:
* Swollen eyes, feet and hands (this condition is commonly known as edema)
* Loss of appetite
* Constant tiredness
* High blood pressure
* Constant feeling of thirst
* Shortness of breath
* Bad breath
* Weight loss
* Nausea and vomiting
* Constant itchiness of the skin
* Muscle cramps
* Tea-colored or cloudy urine
* A yellowish-brown trace on the skin
There are two types of kidney failure, chronic kidney failure or acute kidney failure. Both types can be life-threatening. In both circumstances, the kidneys stop functioning normally and cannot filter wastes or too much water from the blood. Due to this condition, toxic materials start to build up which can cause several complications that can affect different body systems. People who suffer from chronic kidney failure will need kidney transplant or dialysis to be able to survive. If you have kidney failure, or you know someone who has this disease, a strict kidney diet must be followed.
* Drink at least 8-10 glasses of water every day. The main function of your kidneys is to filter toxins from the blood and drain them off through urine. Without adequate amount of water, your kidneys can’t function very well. Generally, most chronic kidney disease and kidney stones are caused by inadequate water intake. You can also eat fruits and vegetables which contain 90 percent of water like cucumber, tomatoes, watermelon and zucchini.
* Eat more vegetables than meat. Too much protein intake can stress your kidneys.
* Avoid foods that have high levels of cholesterol. Eat nondairy products and lean meats instead. Also include whole grains in your kidney diet.
* Avoid goods that are heavily sweetened.
* Quit smoking. Smoking can increase blood pressure which is one of the major causes of kidney disease.
When planning a kidney diet it is still best to consult your physician or a dietician. The right kidney diet plan for you will also depend on the condition that you have. The key to a healthy kidney diet is dedication and the right people to back you up.
About The Publisher:
Kidney is one of the most important organs in our body, And having the right solution for the problem in one’s kidney is very hard to find due to different ideas about it. Kidney Diet have many ways in treating them and we just need to know and to perform them correctly. For more information visit my site at http://mykidneydiet.com/
About Us: http://mykidneydiet.com/
Contact Info: +6519281414
http://www.addpr.com/articles/health_fitness/95704.html
Wednesday, October 19, 2011
Critical evaluation of rituximab rescue in 27 patients with different types of kidney disease.
Minerva Urol Nefrol. 2011 Dec;63(4):263-72.
Critical evaluation of rituximab rescue in 27 patients with different types of kidney disease.
Ganzemueller J, Hartmann B, Keller F, Stracke S.
SourceDepartment of Nephrology, School of Medicine, University of Ulm, Ulm, Germany - frieder.keller@uni-ulm.de.
Abstract
AIM: Rituximab is increasingly being used in the treatment of patients with kidney disease. We evaluated our clinical experience at the Ulm University Hospital.
METHODS: Since 2004, we have administered rituximab as rescue therapy to twenty-seven patients with kidney disease non-responsive to standard treatment. Indications for rituximab were progressive loss of kidney function in thirteen cases; nephrotic syndrome in five cases; humoral rejection after kidney transplantation in five cases and single cases of catastrophic antiphospholipid syndrome (CAPS), pre-emptive removal of ABO incompatible antibodies, pre-transplant removal of panel-reactive antibodies (PRA), and post-transplant lymphoproliferative disease (PTLD). Sixteen patients were treated with both, plasmapheresis and rituximab.
RESULTS: Kidney function recovered in five of thirteen cases. Nephrotic syndrome response was observed in two of five cases. In two of five patients with humoral rejection, kidney transplant function could be preserved. Antiphospholipid antibodies, blood group A antibodies and panel reactive antibodies successfully were reduced, and remission was achieved in the case of the patient with PTLD. Four patients died (15%). Adverse events (N.=10) and infectious complications (N.=15) were most likely due to immunosuppression in general and not to rituximab alone. Toxic leukoencephalopathy was a serious but reversible complication in three cases and occurred particularly after the administration of high-dose rituximab (>375 mg/m2).
CONCLUSION: Rituximab rescue was successful in 48% of our cases (13 of 27). Rituximab did not increase the complication risk of standard immunosuppression. But toxic leukoencephalopathy was identified as a significant rituximab complication.
PMID:21996981[PubMed - in process]
http://www.ncbi.nlm.nih.gov/pubmed/21996981
Critical evaluation of rituximab rescue in 27 patients with different types of kidney disease.
Ganzemueller J, Hartmann B, Keller F, Stracke S.
SourceDepartment of Nephrology, School of Medicine, University of Ulm, Ulm, Germany - frieder.keller@uni-ulm.de.
Abstract
AIM: Rituximab is increasingly being used in the treatment of patients with kidney disease. We evaluated our clinical experience at the Ulm University Hospital.
METHODS: Since 2004, we have administered rituximab as rescue therapy to twenty-seven patients with kidney disease non-responsive to standard treatment. Indications for rituximab were progressive loss of kidney function in thirteen cases; nephrotic syndrome in five cases; humoral rejection after kidney transplantation in five cases and single cases of catastrophic antiphospholipid syndrome (CAPS), pre-emptive removal of ABO incompatible antibodies, pre-transplant removal of panel-reactive antibodies (PRA), and post-transplant lymphoproliferative disease (PTLD). Sixteen patients were treated with both, plasmapheresis and rituximab.
RESULTS: Kidney function recovered in five of thirteen cases. Nephrotic syndrome response was observed in two of five cases. In two of five patients with humoral rejection, kidney transplant function could be preserved. Antiphospholipid antibodies, blood group A antibodies and panel reactive antibodies successfully were reduced, and remission was achieved in the case of the patient with PTLD. Four patients died (15%). Adverse events (N.=10) and infectious complications (N.=15) were most likely due to immunosuppression in general and not to rituximab alone. Toxic leukoencephalopathy was a serious but reversible complication in three cases and occurred particularly after the administration of high-dose rituximab (>375 mg/m2).
CONCLUSION: Rituximab rescue was successful in 48% of our cases (13 of 27). Rituximab did not increase the complication risk of standard immunosuppression. But toxic leukoencephalopathy was identified as a significant rituximab complication.
PMID:21996981[PubMed - in process]
http://www.ncbi.nlm.nih.gov/pubmed/21996981
Sunday, October 02, 2011
Excellent survival after liver transplantation for isolated polycystic liver disease: an European Liver Transplant Registry study.
Transpl Int. 2011 Sep 29. doi: 10.1111/j.1432-2277.2011.01360.x. [Epub ahead of print]
Excellent survival after liver transplantation for isolated polycystic liver disease: an European Liver Transplant Registry study.
van Keimpema L, Nevens F, Adam R, Porte RJ, Fikatas P, Becker T, Kirkegaard P, Metselaar HJ, Drenth JP; for the European Liver and Intestine Transplant Association (ELITA).
Source Department of Gastroenterology and Hepatology, Radboud University Nijmegen Medical Center, Nijmegen, The Netherlands Department of Hepatology, University Hospital KULeuven, Leuven, Belgium Centre Hépato-biliaire, AP-HP Hôpital Paul Brousse, Villejuif, France Department of Surgery, University Medical Center Groningen, Groningen, The Netherlands Department for General, Visceral and Transplant Surgery, Charite Campus Virchow, Universitätsmedizin Berlin, Berlin, Germany Department for General, Visceral and Transplant Surgery, Medizinische Hochschule Hannover, Hannover, Germany Department of Surgery and Transplantation, Rigshospitalet, University of Copenhagen, Denmark Department of Gastroenterology and Hepatology, Erasmus Medical Center, Rotterdam, The Netherlands.
Abstract
Patients with end-stage isolated polycystic liver disease (PCLD) suffer from incapacitating symptoms because of very large liver volumes. Liver transplantation (LT) is the only curative option. This study assesses the feasibility of LT in PCLD. We used the European Liver Transplant Registry (ELTR) database to extract demographics and outcomes of 58 PCLD patients. We used Kaplan-Meier survival analysis for survival rates. Severe abdominal pain (75%) was the most prominent symptom, while portal hypertension (35%) was the most common complication in PCLD. The explantation of the polycystic liver was extremely difficult in 38% of patients, because of presence of adhesions from prior therapy (17%). Karnofsky score following LT was 90%. The 1- and 5-year graft survival rate was 94.3% and 87.5%, while patient survival rate was 94.8% and 92.3%, respectively. Survival rates after LT for PCLD are good.
© 2011 The Authors. Transplant International © 2011 European Society for Organ Transplantation.
PMID:21955068[PubMed - as supplied by publisher]
http://www.ncbi.nlm.nih.gov/pubmed/21955068
Excellent survival after liver transplantation for isolated polycystic liver disease: an European Liver Transplant Registry study.
van Keimpema L, Nevens F, Adam R, Porte RJ, Fikatas P, Becker T, Kirkegaard P, Metselaar HJ, Drenth JP; for the European Liver and Intestine Transplant Association (ELITA).
Source Department of Gastroenterology and Hepatology, Radboud University Nijmegen Medical Center, Nijmegen, The Netherlands Department of Hepatology, University Hospital KULeuven, Leuven, Belgium Centre Hépato-biliaire, AP-HP Hôpital Paul Brousse, Villejuif, France Department of Surgery, University Medical Center Groningen, Groningen, The Netherlands Department for General, Visceral and Transplant Surgery, Charite Campus Virchow, Universitätsmedizin Berlin, Berlin, Germany Department for General, Visceral and Transplant Surgery, Medizinische Hochschule Hannover, Hannover, Germany Department of Surgery and Transplantation, Rigshospitalet, University of Copenhagen, Denmark Department of Gastroenterology and Hepatology, Erasmus Medical Center, Rotterdam, The Netherlands.
Abstract
Patients with end-stage isolated polycystic liver disease (PCLD) suffer from incapacitating symptoms because of very large liver volumes. Liver transplantation (LT) is the only curative option. This study assesses the feasibility of LT in PCLD. We used the European Liver Transplant Registry (ELTR) database to extract demographics and outcomes of 58 PCLD patients. We used Kaplan-Meier survival analysis for survival rates. Severe abdominal pain (75%) was the most prominent symptom, while portal hypertension (35%) was the most common complication in PCLD. The explantation of the polycystic liver was extremely difficult in 38% of patients, because of presence of adhesions from prior therapy (17%). Karnofsky score following LT was 90%. The 1- and 5-year graft survival rate was 94.3% and 87.5%, while patient survival rate was 94.8% and 92.3%, respectively. Survival rates after LT for PCLD are good.
© 2011 The Authors. Transplant International © 2011 European Society for Organ Transplantation.
PMID:21955068[PubMed - as supplied by publisher]
http://www.ncbi.nlm.nih.gov/pubmed/21955068
Tuesday, September 27, 2011
Kidney disease marker predicts who will need dialysis
September 09, 2011|By Shari Roan, Los Angeles Times / For the Booster Shots blogKidney disease affects about 20 million Americans, many of whom end up on dialysis. But there may be a way to identify and treat severe cases earlier in the course of the disease.
In a study published Friday, researchers said that measuring a hormone called FGF-23 can predict which patients will end up needing dialysis.
The hormone, fibroblast growth factor-23, was discovered fairly recently and has attracted a lot of attention from researchers for its crucial role in regulating phosphorus in the body. The new study showed that as kidneys fail, FGF-23 levels rise. Currently, doctors measure phosphorus levels to monitor patients with kidney disease. But FGF-32 appears to be more sensitive and begins to rise well before phosphorus changes are apparent.
"This discovery allows us to predict at-risk patients before they require dialysis," the lead investigator, Dr. Michel Chonchol, an associate professor of nephrology at the University of Colorado School of Medicine, said in a news release. "That's critical because approximately 23% of patients on dialysis die in the first year."
The study appears in the Journal of the American Society of Nephrology.
Return to Booster Shots blog.
Follow me: twitter.com/LATShariRoan
http://articles.latimes.com/2011/sep/09/news/la-heb-kidney-disease-20110909
In a study published Friday, researchers said that measuring a hormone called FGF-23 can predict which patients will end up needing dialysis.
The hormone, fibroblast growth factor-23, was discovered fairly recently and has attracted a lot of attention from researchers for its crucial role in regulating phosphorus in the body. The new study showed that as kidneys fail, FGF-23 levels rise. Currently, doctors measure phosphorus levels to monitor patients with kidney disease. But FGF-32 appears to be more sensitive and begins to rise well before phosphorus changes are apparent.
"This discovery allows us to predict at-risk patients before they require dialysis," the lead investigator, Dr. Michel Chonchol, an associate professor of nephrology at the University of Colorado School of Medicine, said in a news release. "That's critical because approximately 23% of patients on dialysis die in the first year."
The study appears in the Journal of the American Society of Nephrology.
Return to Booster Shots blog.
Follow me: twitter.com/LATShariRoan
http://articles.latimes.com/2011/sep/09/news/la-heb-kidney-disease-20110909
Saturday, September 24, 2011
Pharsight to Work with Critical Path Institute's Polycystic Kidney Disease Consortium on Disease Progression Modeling
Consortium Includes Academic, Regulatory, and Industry Experts in Effort to Accelerate Pace of Medical Product Development
http://www.marketwatch.com/story/pharsight-to-work-with-critical-path-institutes-polycystic-kidney-disease-consortium-on-disease-progression-modeling-2011-09-07
ST. LOUIS, Sep 07, 2011 (BUSINESS WIRE) -- Pharsight, a market-leading provider of software and scientific consulting services to improve productivity and decision-making in clinical drug development, has announced they will be working with Critical Path Institute (C-Path)'s Polycystic Kidney Disease (PKD) Consortium to create and study quantitative models of disease progression in patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD).
The PKD Consortium is a successful collaboration between C-Path, the PKD Foundation, Clinical Data Interchange Standards Consortium (CDISC), and four leading academic medical centers (Tufts University, University of Colorado - Denver, Emory University, and Mayo Clinic). The Consortium is led by C-Path and funded through a grant from the PKD Foundation and philanthropic donations. Additionally, a representative from the U.S. Food and Drug Administration (FDA) serves as an active advisor to the Consortium.
"ADPKD is a debilitating genetic disease affecting more than 600,000 Americans and 12 million people worldwide for which there is currently no known cure or effective treatment," said Ronald Perrone, MD, Co-Director of the PKD Consortium and Professor of Medicine at Tufts University School of Medicine.
"This is a very exciting project that leverages the power of collaboration," said Eslie Dennis, MD, FCP, Executive Director of the PKD Consortium at C-Path. "Traditional endpoints of renal function only show changes very late in the course of the disease, making it difficult to assess the effectiveness of new medications. There is critical need for a biomarker that will assess disease progression at an earlier stage when patients may be more likely to respond to new therapies."
"We are very pleased about this significant scientific partnership with Pharsight," added Jill Panetta, PhD, Interim Chief Executive Officer for the PKD Foundation. "The PKD Consortium has brought together impressive resources in support of our mission to promote research that will lead to the discovery of treatments for PKD and improve the lives of all it affects."
The primary goals of the PKD Consortium are to develop CDISC research data standards for PKD and to use clinical data from PKD patients collected over many years through patient registries and clinical trials to support the FDA-qualification of an imaging biomarker, Total Kidney Volume (TKV), as an endpoint in drug development trials.
Scientists and physicians from Pharsight and the PKD Consortium will use the data collected to develop a quantitative disease progression model that will evaluate the relationship between TKV and the known complications of ADPKD, including rate of loss of kidney function, development of end-stage renal disease, and mortality. These analyses will be used to support the regulatory qualification of TKV as an accepted measure for assessing the progression of ADPKD in clinical trials in which new therapies are tested.
"Critical Path Institute is proud to be the facilitator of this ground breaking collaboration to accelerate the pace of drug development for this disease," said Raymond Woosley, MD, PhD, President and Chief Executive Officer of C-Path. "We welcome the opportunity to work with Pharsight and its team of scientists."
"Pharsight is honored to join this important and innovative project," added JF Marier, PhD, FCP, Vice President and Lead Scientist at Pharsight. "We are all committed to the development of a qualified measure that will encourage drug development organizations to test potential new disease-modifying treatments for ADPKD."
About Pharsight
Pharsight, a Certara(TM) company, is a market-leading provider of software products and scientific consulting services to help pharmaceutical and biotechnology companies improve their drug development process, regulatory compliance and strategic decision-making. Established in 1995, the company's goal is to help customers reduce the time, cost and risk of drug development. With more than 1200 customers worldwide, Pharsight products and services are used by all of the world's top 50 pharmaceutical firms. More information about Pharsight is available at http://www.pharsight.com/ .
About Critical Path Institute (C-Path)
An independent, non-profit organization established in 2005 with public and private philanthropic support from the Southern Arizona community, Science Foundation Arizona (SFAz), and the U.S. Food and Drug Administration (FDA), C-Path is committed to transformational improvement of the drug development process. An international leader in forming collaborations around this mission, C-Path has established first-of-its-kind global partnerships that currently include over 1,000 scientists from government, regulatory agencies, academia, patient advocacy organizations, and thirty major pharmaceutical companies. C-Path is headquartered in Tucson, Arizona, with offices in Phoenix, Arizona, and Rockville, Maryland. More information about C-Path is available at http://www.c-path.org/ .
About The PKD Foundation
Polycystic Kidney Disease affects one in 500 people, regardless of gender, age or ethnicity. More than 600,000 Americans and 12.5 million people worldwide suffer from this debilitating genetic disease. The PKD Foundation is the primary research, education and advocacy group working to find a treatment and a cure for Polycystic Kidney Disease. More information about PKD and the PKD Foundation is available at http://www.pkdcure.org/ .
Registered Trademarks and Trademarks
Pharsight is a registered trademark of Tripos, L.P. All other brands and product names are trademarks or registered trademarks of their respective holders.
SOURCE: Pharsight
Pharsight Contact:
Adam Rutkin, 408-737-6053
Director of Marketing and Business Development
arutkin@pharsight.com
or
Critical Path Institute Contact:
Lisa Romero, 520-547-3440
Communications and Strategic Outreach Manager
lromero@c-path.org
http://www.marketwatch.com/story/pharsight-to-work-with-critical-path-institutes-polycystic-kidney-disease-consortium-on-disease-progression-modeling-2011-09-07
ST. LOUIS, Sep 07, 2011 (BUSINESS WIRE) -- Pharsight, a market-leading provider of software and scientific consulting services to improve productivity and decision-making in clinical drug development, has announced they will be working with Critical Path Institute (C-Path)'s Polycystic Kidney Disease (PKD) Consortium to create and study quantitative models of disease progression in patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD).
The PKD Consortium is a successful collaboration between C-Path, the PKD Foundation, Clinical Data Interchange Standards Consortium (CDISC), and four leading academic medical centers (Tufts University, University of Colorado - Denver, Emory University, and Mayo Clinic). The Consortium is led by C-Path and funded through a grant from the PKD Foundation and philanthropic donations. Additionally, a representative from the U.S. Food and Drug Administration (FDA) serves as an active advisor to the Consortium.
"ADPKD is a debilitating genetic disease affecting more than 600,000 Americans and 12 million people worldwide for which there is currently no known cure or effective treatment," said Ronald Perrone, MD, Co-Director of the PKD Consortium and Professor of Medicine at Tufts University School of Medicine.
"This is a very exciting project that leverages the power of collaboration," said Eslie Dennis, MD, FCP, Executive Director of the PKD Consortium at C-Path. "Traditional endpoints of renal function only show changes very late in the course of the disease, making it difficult to assess the effectiveness of new medications. There is critical need for a biomarker that will assess disease progression at an earlier stage when patients may be more likely to respond to new therapies."
"We are very pleased about this significant scientific partnership with Pharsight," added Jill Panetta, PhD, Interim Chief Executive Officer for the PKD Foundation. "The PKD Consortium has brought together impressive resources in support of our mission to promote research that will lead to the discovery of treatments for PKD and improve the lives of all it affects."
The primary goals of the PKD Consortium are to develop CDISC research data standards for PKD and to use clinical data from PKD patients collected over many years through patient registries and clinical trials to support the FDA-qualification of an imaging biomarker, Total Kidney Volume (TKV), as an endpoint in drug development trials.
Scientists and physicians from Pharsight and the PKD Consortium will use the data collected to develop a quantitative disease progression model that will evaluate the relationship between TKV and the known complications of ADPKD, including rate of loss of kidney function, development of end-stage renal disease, and mortality. These analyses will be used to support the regulatory qualification of TKV as an accepted measure for assessing the progression of ADPKD in clinical trials in which new therapies are tested.
"Critical Path Institute is proud to be the facilitator of this ground breaking collaboration to accelerate the pace of drug development for this disease," said Raymond Woosley, MD, PhD, President and Chief Executive Officer of C-Path. "We welcome the opportunity to work with Pharsight and its team of scientists."
"Pharsight is honored to join this important and innovative project," added JF Marier, PhD, FCP, Vice President and Lead Scientist at Pharsight. "We are all committed to the development of a qualified measure that will encourage drug development organizations to test potential new disease-modifying treatments for ADPKD."
About Pharsight
Pharsight, a Certara(TM) company, is a market-leading provider of software products and scientific consulting services to help pharmaceutical and biotechnology companies improve their drug development process, regulatory compliance and strategic decision-making. Established in 1995, the company's goal is to help customers reduce the time, cost and risk of drug development. With more than 1200 customers worldwide, Pharsight products and services are used by all of the world's top 50 pharmaceutical firms. More information about Pharsight is available at http://www.pharsight.com/ .
About Critical Path Institute (C-Path)
An independent, non-profit organization established in 2005 with public and private philanthropic support from the Southern Arizona community, Science Foundation Arizona (SFAz), and the U.S. Food and Drug Administration (FDA), C-Path is committed to transformational improvement of the drug development process. An international leader in forming collaborations around this mission, C-Path has established first-of-its-kind global partnerships that currently include over 1,000 scientists from government, regulatory agencies, academia, patient advocacy organizations, and thirty major pharmaceutical companies. C-Path is headquartered in Tucson, Arizona, with offices in Phoenix, Arizona, and Rockville, Maryland. More information about C-Path is available at http://www.c-path.org/ .
About The PKD Foundation
Polycystic Kidney Disease affects one in 500 people, regardless of gender, age or ethnicity. More than 600,000 Americans and 12.5 million people worldwide suffer from this debilitating genetic disease. The PKD Foundation is the primary research, education and advocacy group working to find a treatment and a cure for Polycystic Kidney Disease. More information about PKD and the PKD Foundation is available at http://www.pkdcure.org/ .
Registered Trademarks and Trademarks
Pharsight is a registered trademark of Tripos, L.P. All other brands and product names are trademarks or registered trademarks of their respective holders.
SOURCE: Pharsight
Pharsight Contact:
Adam Rutkin, 408-737-6053
Director of Marketing and Business Development
arutkin@pharsight.com
or
Critical Path Institute Contact:
Lisa Romero, 520-547-3440
Communications and Strategic Outreach Manager
lromero@c-path.org
Thursday, September 08, 2011
Scientists explore new model in battle against kidney disease
September 8, 2011 — 6:31am ET | By Ryan McBride
A multi-organizational effort to combat polycystic kidney disease has turned to the scientists at software and consulting firm Pharsight to develop quantitative models of the disorder. It's another step in the Critical Path Institute's PKD Consortium quest to advance CDISC research data standards and an imaging biomarker for the kidney disease, which affects one in 500 Americans and more than 12 million people worldwide.
The group wants to develop the imaging biomarker, known as total kidney volume (TKV), and gain the FDA's support for the marker as a key measurement of autosomal dominant polycystic kidney disease in clinical trials for drugs against the genetic condition. Toward that end, Pharsight and the PKD Consortium aim to create a disease-progression model with data collected from studies of the ailment "that will evaluate the relationship between TKV and the known complications of ADPKD, including rate of loss of kidney function, development of end-stage renal disease and mortality," Pharsight said in a release.
"This is a very exciting project that leverages the power of collaboration," said Dr. Eslie Dennis, executive director of the PKD Consortium, in a statement. "Traditional endpoints of renal function only show changes very late in the course of the disease, making it difficult to assess the effectiveness of new medications. There is critical need for a biomarker that will assess disease progression at an earlier stage when patients may be more likely to respond to new therapies."
The groups involved in the consortium also include the PKD Foundation, a financial supporter of the effort, the Clinical Data Interchange Standards Consortium (CDISC), as well as experts from Emory University, the Mayo Clinic, Tufts University and the University of Colorado, Denver.
- here's the release
Read more: Scientists explore new model in battle against kidney disease - FierceBiotechIT http://www.fiercebiotechit.com/story/scientists-explore-new-model-battle-against-kidney-disease/2011-09-08#ixzz1XMYxHYTb
Subscribe: http://www.fiercebiotechit.com/signup?sourceform=Viral-Tynt-FierceBiotechIT-FierceBiotechIT
A multi-organizational effort to combat polycystic kidney disease has turned to the scientists at software and consulting firm Pharsight to develop quantitative models of the disorder. It's another step in the Critical Path Institute's PKD Consortium quest to advance CDISC research data standards and an imaging biomarker for the kidney disease, which affects one in 500 Americans and more than 12 million people worldwide.
The group wants to develop the imaging biomarker, known as total kidney volume (TKV), and gain the FDA's support for the marker as a key measurement of autosomal dominant polycystic kidney disease in clinical trials for drugs against the genetic condition. Toward that end, Pharsight and the PKD Consortium aim to create a disease-progression model with data collected from studies of the ailment "that will evaluate the relationship between TKV and the known complications of ADPKD, including rate of loss of kidney function, development of end-stage renal disease and mortality," Pharsight said in a release.
"This is a very exciting project that leverages the power of collaboration," said Dr. Eslie Dennis, executive director of the PKD Consortium, in a statement. "Traditional endpoints of renal function only show changes very late in the course of the disease, making it difficult to assess the effectiveness of new medications. There is critical need for a biomarker that will assess disease progression at an earlier stage when patients may be more likely to respond to new therapies."
The groups involved in the consortium also include the PKD Foundation, a financial supporter of the effort, the Clinical Data Interchange Standards Consortium (CDISC), as well as experts from Emory University, the Mayo Clinic, Tufts University and the University of Colorado, Denver.
- here's the release
Read more: Scientists explore new model in battle against kidney disease - FierceBiotechIT http://www.fiercebiotechit.com/story/scientists-explore-new-model-battle-against-kidney-disease/2011-09-08#ixzz1XMYxHYTb
Subscribe: http://www.fiercebiotechit.com/signup?sourceform=Viral-Tynt-FierceBiotechIT-FierceBiotechIT
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