Rare Kidneys Connect – Baltimore January 25, 2024 by Hillary Kent Join NephCure, along with fellow patients and caregivers affected by rare kidney diseases, in Baltimore for an informative session on protein-spilling kidney conditions and strategies for their effective management. This event is FREE and a low-sodium snack will be provided! We extend a warm invitation to individuals impacted by the following rare kidney diseases to participate: Focal segmental glomerulosclerosis (FSGS) Nephrotic syndrome IgA nephropathy (IgAN) Membranous nephropathy (MN) Minimal change disease (MCD) C3 glomerulonephritis (C3G)/membranoproliferative glomerulonephritis (MPGN) IgM nephropathy (IgMN) Together, we will delve into various topics, including: Education and management of these diseases Advances in treatments and ongoing research Insights from a patient panel on coping with rare kidney diseases Access to patient resources
NephCure Champion Spotlight: Nicholas Ariyo January 25, 2024 by Hillary Kent When I was 17, I was diagnosed with kidney disease called FSGS. This was 1 year after my little brother had been diagnosed with the same thing. While my brother was able to be managed on medication, mine was more malignant. It was not explained well to me at the time so it was hard for my family and I to figure out why it was happening and what I did wrong to cause this. I was placed on heavy steroids and Myfortic. Due to the side effects of the steroids, I had a difficult time during my senior year of high school. Once I graduated, I went to Texas Tech to try and enter their fast-track medical school program. This was when I started to experience kidney failure. I was in denial about my symptoms and was thinking the swelling and fatigue were side effects of the medications. I was so swollen my skin would literally tear open and streams of water would come out. It was only when my mom and sister begged me to go to the college clinic that I went and was told it was a miracle I was still alive and that I needed to go to the emergency room immediately. I flew back home and when my parents saw how swollen I was they bursted into tears. I was taken to the ER where they placed an emergency catheter to begin dialysis. I was told I had 40 liters of extra fluid in my body. I was now on hemodialysis 3 times a week for a few months before transferring to peritoneal dialysis. I immediately re-enrolled at the University of Houston. Between the adjustments to my life, school, and work, I was not keeping up with my treatments correctly, which led me to having a seizure in my dorm room, falling and hitting my head on the corner of my desk. Luckily, I was rooming with my little brother so he was able to call an ambulance immediately. I was in a coma and the doctors said they had no idea if or when I would wake up. After three days, I woke up from the coma. Between this and college I was placed on probation at the University of Houston. I then transferred to Texas Southern University to finish my last year of college. After graduating, I was accepted into the Texas Southern University college of pharmacy where I also received my kidney transplant after 5 years on dialysis. I was told to withdrawal from school due to being behind in courses and needing to recover due to my health. But, fortunately my body healed quicker than expected and I was able to catch up and finish the semester on the Dean’s list. A couple of years later my little brother progressed to kidney failure and then had to begin hemodialysis. He was not living with my parents at this time so between my mom and I, we would take him to his treatments. He then changed to peritoneal dialysis. After a few months and issues with his dialysis treatment he passed away in his apartment. I still feel guilty about not being able to provide him with more support during that time. This is why shedding light on the effects of rare kidney disease is a passion, not a pastime. I want to help others who were not as fortunate and blessed as I was to make it through so many obstacles and hardships, in whatever way possible. — Nicholas Ariyo, 4th year Pharm.D candidate at TSU College of Pharmacy
NephCure Funded Research 2022 2022 NephCure Award Recipients NEPTUNE Grant Awardees Ana C. Onuchic-Whitford, MD (Brigham and Women’s Hospital) Discovering the landscape of allele-specific expression in the human kidney Ana Claudia Onuchic-Whitford, MD, is a nephrologist at Brigham and Women’s Hospital and an Instructor in Medicine at Harvard Medical School in Boston, MA. She received her M.D. from the University of São Paulo in Brazil, where she also worked in a basic oncology laboratory and completed residency in Internal Medicine. After relocating to the U.S., she repeated her Internal Medicine residency at the University of Connecticut and completed a five-year clinical and research fellowship in adult nephrology in the joint Brigham and Women’s Hospital / Massachusetts General Hospital program. Impressed by how genetics revolutionized medicine and motivated to find missing diagnoses for kidney disease patients, she became interested in searching for genetic determinants of kidney disorders. From a research standpoint, Ana joined Boston Children’s Hospital (BCH) as a post-doctoral research fellow to study the genetics of nephrotic syndrome and chronic kidney disease of unknown etiology in the Hildebrandt lab. Her current research in computational genomics with the Sampson Lab at BCH aims to identify imbalances in gene expression and find novel mechanisms of gene regulation that can contribute to glomerular disease. From a clinical perspective, Ana helped establish the Kidney Genetics & PKD Clinic at Brigham and Women’s Hospital. She now leads this clinic and directly cares for over 100 patients with genetic kidney diseases, including genetic FSGS, Alport Syndrome, polycystic kidney disease, and congenital kidney anomalies, among other inherited nephropathies. In addition to advancing the field of glomerular diseases and nephrotic syndrome, the potential to bridge the science to both genetics-driven patient care and to education in genomic literacy is a valued aspect of her research in computational genomics. Lay Summary of the Project: Description: During the genetic revolution of the last two decades, important discoveries have revealed the major role of genetic factors in kidney function, development and disease. Humans have tens of thousands of genes, which are segments of DNA that guide how our bodies function. Hundreds of forms of chronic kidney disease have been shown to be caused by defects in single genes – including more than 60 genes for nephrotic syndrome (NS), a clinical condition in which the kidneys lose a large amount of protein. As genetic knowledge has transformed the understanding of kidney disease, we are moving to personalized, genetics-guided clinical kidney care. However, many patients with suspected genetic kidney disease still do not have a diagnosis, and the mechanisms by which some genetic factors cause disease are still obscure. In this setting, different genetic studies are needed, including one called allele-specific expression (ASE). An allele is a copy of a gene: for almost all of our genes, there are two copies (two alleles) – one that we received from our mother, and one received from our father. The DNA sequence of a gene is used to create mRNA, a molecule that serves as an intermediate for the subsequent production of a protein. By analyzing someone’s mRNA sequences, we can often identify specifically from which of the two gene copies (two alleles) the mRNA molecules originated from. When performing ASE analysis, we calculate the number of mRNA molecules (collectively called “gene expression”) originating from each of the two alleles: if the number is the same, this is called balanced expression. However, if most of the mRNA comes from one specific allele, this is called “allele-specific expression.” ASE has already been shown to contribute to several illnesses, including autism, heart disease and cancer. For example, in a person who has one normal gene copy and one defective gene copy, if the expression is skewed towards the defective allele, this person may have more severe disease. From a kidney standpoint, a prior study by our group found that individuals with NS had ASE of NPHS1 – a gene responsible for the production of nephrin, a very important component of the kidney filter. However, ASE studies in human kidney disease are limited. Goals and Significance: In this project, our goal is to identify occurrence of ASE in the kidney filtering units (glomeruli) of individuals from the Nephrotic Syndrome Study Network (NEPTUNE). We believe that discovering skewed expression of certain genes can help identify new genetic mechanisms contributing to kidney disease, urine protein loss and NS. To this end, we will search for ASE of genes known to cause NS, when mutated. We will then look for genes that have greater ASE in NEPTUNE participants, compared to healthy kidney samples. Next, we will search for which locations within the DNA are causing the imbalance in mRNA. Finally, we will overlap this with a database that shows which DNA regions are actively being used by each kidney cell type. This can allow us to pinpoint where in the kidney the ASE is happening. In summary, our study seeks to identify ASE occurring in kidneys of patients with significant urine protein loss (e.g., NS) and use this data to identify new pathways leading to kidney disease. We also aim to make this ASE information available to all researchers through an online portal, to contribute to more studies and, hopefully, to future clinical diagnostic and treatment strategies. CureGN Pilot Project Grant Awardees Rosemary V. Sampogna, M.D., PhD & Simone Sanna-Cherchi, M.D. (Columbia University Irving Medical Center/Vagelos College of Medicine in New York) Patient-derived kidney organoids to study mechanisms and therapeutics in Mendelian forms of FSGS Rosemary V. Sampogna, M.D., Ph.D. Dr. Sampogna is an Associate Professor in the Division of Nephrology at Columbia University Irving Medical Center / Vagelos College of Medicine in New York. Her research is focused on the molecular and genetic mechanisms that specify kidney architecture and ultimately determine function, on the role of cadherins during nephrogenesis, and on the role of the circadian clock as a developmental timer that determines the rate of branching and organogenesis. More recently, her work has been directed at studying the functional consequences of human mutations using vertebrate and cellular models, including patient-derived induced pluripotent stem cells (iPSC) and organoids. Simone Sanna-Cherchi, M.D. Dr. Sanna-Cherchi is an Associate Professor in the Division of Nephrology at Columbia University Irving Medical Center / Vagelos College of Medicine in New York. His main area of research involves the genetics of rare forms of kidney diseases and their complications, with special focus on congenital anomalies of the kidney and urinary tract (CAKUT) and nephrotic syndrome caused by minimal change disease (MCD) and focal segmental glomerulosclerosis (FSGS). His work spans the entire spectrum of genomics research, including genome-wide association studies and large-scale sequencing approaches for rare variants association. To complement his sequencing and genotyping approaches for human traits, he has developed new mouse models for congenital defects of the urinary tract and nephrotic syndrome. With the support of the NephCure – CureGN Pilot Project Grant Award we plan to generate a panel of iPSCs derived from CureGN participants who carry mutations in FSGS Mendelian genes. These cell lines will allow us to generate patient-specific kidney organoids. These models will allow to study the effect of mutations of key regulatory genes for podocyte development, structure, and function in the patient’s genomic and epigenomic context. We hope that our studies will help develop new therapeutic approaches for these devastating conditions. Update: Dr. Sanna-Cherchi and Dr. Sampogna, with this support, have generated patient-derived induced pluripotent stem cells (iPSCs) from children and young adults carrying causal variants in FSGS-associated genes such as TRIM8, TRPC6 and others. They are studying the effect of these variants in iPSC-derived 3D kidney organoids using a multiomic approach that leverages on simultaneous interrogation of single nucleus transcriptomic and open chromatin signatures, coupled with proteomics studies. The ultimate goals of this project are to identify gene-specific molecular mechanisms that might lead to precise drug targeting approaches. See Awardees From Other Years: 2021 2022 2020 2019 2018 2017 2016 2015 2014 2013 2012 2011 2009 2008
Rare Kidney Connect – Los Angeles January 8, 2024 by Hillary Kent 1 Comment Join NephCure and other rare kidney disease patients and caregivers on Saturday, February 3, 2024, from 1:00 to 4:30 p.m. in Los Angeles for a FREE, in-person educational event to learn more about rare, protein-spilling kidney diseases and how to best manage them. Plus, enjoy a low-sodium snack and connect with other rare kidney disease patients in your community. We invite everyone affected by the following rare kidney diseases to attend: Focal segmental glomerulosclerosis (FSGS) Nephrotic syndrome IgA nephropathy (IgAN) Membranous nephropathy (MN) Minimal change disease (MCD) C3 glomerulonephritis (C3G)/membranoproliferative glomerulonephritis (MPGN) IgM nephropathy (IgMN) This event will cover topics such as disease education and management, progress in treatments and research, and patient resources. You’ll also hear from a patient panel on coping with rare kidney disease! Register today!
NephCure Applauds the Introduction of Federal Legislation to Improve Kidney Patient Access to Early Interventions, Improved Diagnostics & Access to Treatments December 15, 2023 by Hillary Kent Washington, D.C., December 15, 2023 – NephCure, a leading national patient advocacy organization dedicated to accelerating research and finding better treatments for rare kidney diseases, today applauded the introduction of the New Era of Preventing End-Stage Kidney Disease Act H.R.6790 in the U.S. House of Representatives. The legislation is championed by Representatives Gus Bilirakis (R-FL) and Terri Sewell (D-AL) and represents a crucial step forward in our nation’s efforts to address rare kidney diseases through research, better diagnostics, and physician and patient education. The New Era of Preventing End-Stage Kidney Disease Act (“New Era Act”) is bipartisan federal legislation that will improve outcomes and quality of life for hundreds of thousands of Americans living with rare kidney disease (RKD) and their families. This legislation will support early intervention, improve access to better treatments, and reduce the physical, psychological, social, and economic impact of RKD through research, better diagnostics, and physician and patient education. The legislation will help mitigate the burden of rare kidney diseases on patients, families, and the health care system, laying the foundation for a new paradigm of proactive and effective kidney disease care. “Our life-saving legislation will help remove diagnostic and treatment barriers for many patients suffering with a rare disease,” said Congressman Gus Bilirakis. “Through the establishment of Rare Kidney Disease Research Centers of Excellence and increased provider education efforts, we will empower providers to better identify the signs and symptoms of rare kidney disease, which will lead to improved treatment options and better patient outcomes.” “Far too many people living with rare kidney disease have trouble finding specialized care providers. Increasing awareness and education is crucial to caring for rare kidney disease patients, which is why I’m so proud to introduce the New Era For Preventing End Stage Kidney Disease Act. This legislation will make critical improvements to the way patients with rare kidney disease, especially those in underserved communities, access and receive care,” said Rep. Sewell. “NephCure commends Congressman Gus Bilirakis (R-FL) and Congresswoman Terri Sewell (D-AL) for championing the New Era of Preventing End-Stage Kidney Disease Act and efforts to see a future in which improved diagnoses, access to treatments, and patient empowerment converge to reshape the trajectory of rare kidney diseases,” said NephCureCEO Joshua Tarnoff. “This bill can change how we take care of rare kidney disease patients through earlier detection and access to the right treatments, providing rare kidney disease education opportunities to doctors and patients, and allocating money for research. We are proud of the essential contributions the rare kidney disease community played in the development and introduction of the New Era of Preventing End-Stage Kidney Disease Act. We are committed to continue working alongside Congressman Bilirakis and Congresswoman Sewell to pass this important legislation in the 118th Congress.” The New Era Act: A New Approach to Improve the State of Kidney Care Reduce Kidney Failure. Mandates the U.S. Department of Health & Human Services (HHS) to evaluate treatment methods that would delay or eliminate the need for dialysis and transplant and provide legislative recommendations to Congress to support its findings. Close the Gap for Underserved Communities. Directs HHS to make recommendations that would improve care in communities that have disproportionate rates of RKD. HHS will study a range of issues relating to early intervention, testing, and treatment, including access to kidney doctors, patient trust of their health care provider, and the utility, impact, and barriers associated with genetic testing. Advance Research and Standard of Care. Creates regional Centers of Excellence on Rare Kidney Disease Research at the National Institutes of Health (NIH) which will support research, public awareness, and resources which could lead to innovative, less invasive treatments and possibly a cure for rare kidney diseases. Enhance Provider Education. Establishes nephrology fellowships and provides continuing education and primary care training on RKD diagnoses and treatment. This training will enhance physician knowledge and increase the number of experts available to patients. Empower Patients & Communities. The New Era Act will support public information and patient education campaigns, promoting informed communities and empowering patients to take charge of their health care journey. Read the full text of H.R.6790 by clicking here. NephCure encourages all of its community members to take action now by urging their Members of Congress to support the New Era legislation. By the Numbers: The State of Kidney Care Top 10 leading causes of death in the U.S. include kidney disease. One in seven adults live with chronic kidney disease. Nine in ten adults with chronic kidney disease are unaware of their condition. The incidence of Focal segmental glomerulosclerosis (FSGS), a rare kidney disease, is around 5 times higher in Black patients when compared to white patients. The annual cost for Medicare to treat kidney failure is $124.5 billion, which is driven by rare and chronic kidney disease. Patients on dialysis spend around 12 hours a week connected to medical devices. About NephCure NephCure is a leading national patient advocacy organization dedicated to empowering people with rare, protein-spilling kidney disease to take charge of their health, while leading the revolution in research, new treatments, and care. NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now new treatments and more than 60 interventional drug trials for rare kidney disease.
New Era of Preventing End-Stage Kidney Disease Act H.R. 6790 Take action by asking Congress to support the New Era Act, using our prefilled automated system. It only takes a minute. CLICK HERE TO TAKE ACTION NOW About the Bill The New Era of Preventing End-Stage Kidney Disease Act (“New Era Act”), introduced by Representatives Gus Bilirakis (R-FL) and Terri Sewell (D-AL), has the potential to bring about significant positive changes in how we understand, treat, and study rare kidney diseases (RKD). This legislation aims to help people with RKD by finding and treating diseases earlier, enhancing education of doctors and patients, and conducting more research. This could lessen the impact of RKD on people’s lives and reduce costs for patients and the health care system in the long run. Reduce Kidney Failure. The New Era Act will require the U.S. Department of Health & Human Services (HHS) to evaluate treatment methods that would delay or eliminate the need for dialysis and transplant and provide legislative recommendations to Congress to support its findings. The Act would also require a report to Congress on how to slow RKD progression, including strategies to increase the use of routine urinalysis and genetic testing. Close the Gap for Underserved Communities. The New Era Act directs HHS to make recommendations that would improve care in communities that have disproportionate rates of RKD. HHS will study a range of issues relating to early intervention, testing, and treatment, including access to kidney doctors, patient trust of their health care provider, and the utility, impact, and barriers associated with genetic testing, in particular for the APOL1 gene mutations (found only in individuals of African descent and associated with greatly increased rates of non-diabetic kidney disease). Advance Research and Standard of Care. The New Era Act will create regional Centers of Excellence on Rare Kidney Disease Research at the National Institutes of Health (NIH) which will support research, public awareness, and resources which could lead to innovative, less invasive treatments and possibly a cure. These centers would be able to share expertise that could improve uniform standards of care, reducing the likelihood for poor or variable health outcomes among RKD patients. Enhance Provider Education. The New Era Act establishes nephrology fellowships and provides continuing education and primary care training on RKD diagnoses and treatment. This training will enhance physician knowledge and increase the number of experts available to patients. Empower Patients & Communities. The New Era Act will support public information and patient education campaigns, promoting informed communities and empowering patients to take charge of their health care journey. Led by Community Action Revolutionizing rare kidney disease treatment has been a longstanding priority for NephCure. In 2020, NephCure and its partner organizations facilitated the first-ever Rare Kidney Disease Roundtable and resulting white paper entitled “We Deserve Better: Revolutionizing Rare Kidney Disease,” which outlined urgent needs in the rare kidney disease space, specifically for patients and their families. Based on these community insights, NephCure worked closely with champions in the House of Representatives on the introduction of the New Era of Preventing End-Stage Kidney Disease Act in the 117th Congress, garnering bipartisan support. NephCure hosted its Rare Kidney Disease (RKD) Week in July 2023 to raise awareness and advocate for legislative change to improve the lives of RKD patients and their families. A record 61 advocates from 20 states flew to Washington, D.C., for the annual Rare Kidneys on the Hill Day, making it NephCure’s largest Hill Day to date. Advocates and NephCure staff conducted over 70 in-person meetings with staff from congressional offices in the U.S. House of Representatives and U.S. Senate and hosted a briefing on the New Era of Preventing End-Stage Kidney Disease Act. Now, NephCure is working with Congress to advance the New Era of Preventing End-Stage Kidney Disease Act in the 118th Congress as our key legislative priority. NephCure has collaborated with rare kidney patients, stakeholder organizations and advocates across the country to drive this important legislation forward, advocating for its passage and implementation to make a positive impact on the lives of those affected by rare kidney diseases. In line with our unwavering dedication to leading the revolution in research, new treatments, and care, NephCure is committed to ensuring that the New Era Act becomes law, as it holds the potential to significantly improve the lives of individuals grappling with rare kidney diseases. The Urgency for Action Kidney disease affects 37 million individuals in the U.S., posing significant health and financial burdens. Patients often face challenges such as delayed and inaccurate diagnoses, limited treatment options, and a lack of access to medical specialists. These hurdles can lead to the progression of kidney disease to end-stage, requiring dialysis or transplantation – treatments that impact the quality of life of patients and strain health care resources. Diagnosis. Gaps in early, accurate detection programs for chronic and rare kidney disease lead to higher health care costs and worse health outcomes. Treatment. The lack of FDA-approved and cost-effective therapies for RKD leaves patients without options to treat their disease. Over 90% of rare diseases have no cure. Specialized Care. Patients with kidney disease face barriers to access specialized care, which negatively impacts their health, treatment costs, and quality of life. By the Numbers: The State of Kidney Care Over 37 million Americans are affected by kidney disease. Kidney disease ranks as the tenth leading cause of death in the United States. One in seven adults live with chronic kidney disease. There are 150 different rare kidney diseases affecting hundreds of thousands of patients. The incidence of focal segmental glomerulosclerosis (FSGS), a rare kidney disease, is around 5 times higher in Black patients when compared to white patients. The annual cost for Medicare to treat kidney failure is $124.5 billion, which is driven by rare and chronic kidney disease. Patients on dialysis spend around 12 hours a week connected to medical devices. Take Action Today Take action by asking Congress to support the New Era Act, using our prefilled automated system. It only takes a minute. CLICK HERE TO TAKE ACTION NOW Together, we can make a difference and shape a future in which rare kidney diseases are accurately diagnosed, innovative treatments are accessible, and patients are empowered to take control of their health. Join us in supporting the New Era Act and be a part of the effort to enhance kidney disease care. Contact your Members of Congress to express your support for this transformative legislation. Spread the word about the New Era Act on social media using #NewEraAct. Stay connected with NephCure for updates and opportunities to get involved.
NephCure Champion Spotlight: Jazrome “AJ” Coulter July 6, 2023 by Kylie Karley Jazrome was diagnosed at the age of 29. The onset of the diseased started when he was 27, resulting in him to be hospitalized for irregular heartbeat. His blood pressure at the time was 234/176 when recorded from his right arm, yet 99/78 when placed on his left arm. Initially, the doctors were going to discharge him. However, the medical team ordered a kidney biopsy that resulted in him being then diagnosed with stage 4 kidney disease. A year later his kidneys would fail and Jazrome spent six years on dialysis. This information is vital to his story: he didn’t discover he had FSGS until his third year of dialysis. Jazrome remembers playing football in college and at the time weighing 250 lbs and could bench 585 pounds! Just to give you an idea of his size and the toll dialysis took on his body— today he weighs 165 pounds (170 pounds on a cheat day). He would give plasma during the week and the doctors would tell him that he was spilling protein. Jazrome never thought anything of it, until it started affecting his football career. He was only 22. Jazrome reports that due to his lack of knowledge regarding his diagnosis, he has so much respect for organizations like NephCure. “Education and research n truly save lives. While also allowing patients and caregivers to live more comfortable lives. I had very little understanding of FSGS. Maybe, if I had the resources that are offered today, I could have avoided the transition from stage 4 to stage 5,” Jazrome said. He recalls one summer he went to the hospital 26 consecutive times and at one point he suffered from a collapsed lung. However, last year, Jazrome received “the gift of life,” a new kidney! But he still has endure kidney-related issues as well as numerous medications and side-effects. Jazrome is currently a touring comedian and works with 7 kidney organizations worldwide! He also has a non-profit named Diseased Jokes where he builds comedy benefits and raises money for different chronic-illness organizations. He is also the self-proclaimed “kidney comic” and wants to raise more awareness about FSGS. In his comedy set, he educates, motivates, and gives hope through humor. To help continue to raise awareness about rare kidney disease, share your story with NephCure here.
NephCure Unveils New Branding March 31, 2023 by Kylie Karley Reflecting Organization’s Focus on Rare Kidney Disease KING OF PRUSSIA, PA (March 31, 2023) — NephCure, formerly NephCure Kidney International, announced Friday a new visual and written brand more distinctly centered around the rare kidney disease (RKD) community they serve. With nearly a year of research and development behind it, this new brand more accurately reflects the nonprofit’s unique position in the RKD space, as well as the explosive growth and transformation the RKD field has seen over the past several years. With this incredible growth, NephCure has entered a new era, and now has the opportunity to reach and support more people affected by RKD across the world. Through robust research, the patient advocacy organization discovered how critical it is to distinguish their brand, allowing those who may be new to the community to understand NephCure’s direct connection to RKD and collective goal to find a cure. “We are extremely proud to unveil our new branding. It not only represents the wave of innovation in the rare kidney disease space that NephCure, researchers, industry partners, and other key stakeholders have helped cultivate together, but also encapsulates the incredibly special community fostered at NephCure,” said NephCure’s CEO, Josh Tarnoff. “We are on the cusp of monumental breakthroughs in rare kidney disease research. New treatments and innovations are upon us, and our updated branding now conveys this cutting-edge shift taking place.” Unique among the major kidney organizations, NephCure is squarely focused on nephrotic syndrome, FSGS, IgAN, and other rare, protein-spilling kidney diseases. NephCure acts as the connector amongst patients and families, healthcare providers, government agencies, industry partners, and other key stakeholders, while also guiding patients to the best care and treatment options possible. “We’ve listened to you — our valued community of patients, caregivers, volunteers, researchers, and other key supporters — through extensive rounds of interviews, surveys, and analysis to ensure that everyone who is a part of our community feels represented, understood, and, ultimately, inspired for progress,” said Kylie Karley, NephCure’s Marketing & Communications Director. Initial elements of NephCure’s brand evolution include: New Logo: The new NephCure logo represents community and togetherness. Featuring a modern spiral of cascading lines that converge together to form a kidney, it demonstrates NephCure’s focus on facilitating connections within the RKD community. This shape also doubles as a “spark,” representing NephCure’s position of leadership and innovation in the nephrology field as they work to advance rare kidney disease research toward more effective treatments. Shortened Name: Through comprehensive research, it was discovered that “NephCure” by and large was the most prevalent name people used to refer to the organization. Since there was high awareness and equity in this specific part of the former name, they decided to officially shorten it to simply “NephCure,” while also developing a supporting tagline to further clarify their mission. New Tagline: “For rare kidney disease” reflects NephCure’s distinct focus on RKD, as well as their passion and focus to continuously strive to find a cure. New Vision Statement: “A world where all who are affected by rare, protein-spilling kidney disease are connected to new and better treatments — and one day, a cure.” New Mission Statement: “To empower people with rare, protein-spilling kidney disease to take charge of their health, while leading the revolution in research, new treatments, and care.” For the second phase of NephCure’s rebranding work, the organization plans to update its website, NephCure.org, to reflect the new brand updates that have gone into effect today. NephCure will continue to update its community on the status of this website redesign. To learn more about NephCure’s new visual and written branding, visit brand.nephcure.org. About NephCure: NephCure’s mission is to empower people with rare, protein-spilling kidney disease to take charge of their health, while leading the revolution in research, new treatments, and care. Founded in 2000 by a group of committed patient parents, NephCure has invested more than $40 million in kidney disease research and helped create a landscape where there are now new treatments and more than 60 interventional drug trials for rare kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity. ###
NephCure Action Network Members Help Enact Sections of New Era Bill into Law March 3, 2023 by Kylie Karley NephCure thanks our NephCure Action Network (NCAN) members for helping to make our end-of-year legislative accomplishments possible. As 2022 and the 117th Congress drew to an end, Congress passed its fiscal year (FY) 2023 funding through an omnibus package, which included some hard-fought NephCure policy priorities. The key to this accomplishment was the collaborative effort between the advocacy team, external partners, and most of all, NephCure’s community of stakeholders — including patient volunteers, families, health care professionals, and researchers. Their meetings, phone calls, letters, and other grassroots activities helped educate lawmakers about rare kidney disease (RKD) and the need for improved funding for research, treatments, and health care services. Within the omnibus package, focal segmental glomerulosclerosis (FSGS) and nephrotic syndrome were included as eligible conditions to participate in the Department of Defense Peer-Reviewed Medical Research Program (DOD PRMRP). The DOD PRMRP was established in 1999 to support research across the full range of science and medicine, with an underlying goal of enhancing the health, care, and wellbeing of military service members, veterans, retirees, and their family members. It continues to serve as an additional avenue for accelerating critical research on rare kidney diseases for the development of lifesaving treatments for patients. The omnibus package also included more than $120 billion to fund initiatives supported by NephCure, including organ transplantation, chronic disease education, and minority health and health disparities, among others. In addition to these wins, two sections taken from the New Era for Preventing End-Stage Kidney Disease Act, which was introduced in Congress last year, were also included and enacted into law! In the first section, “Diversity in National Institutes of Health (NIH) Kidney Disease Research Populations,” the Appropriations Committee directs the NIH to submit an update on its research related to kidney disease, including research focusing on health disparities in the prevention, diagnosis, and treatment of kidney disease among racial and ethnic minority populations, in the FY 2024 Congressional Justification. The second section, “Rare Kidney Diseases in Health Equity Initiatives,” requests the United States Department of Health and Human Services (HHS) to provide an update on disparities in kidney care and the inclusion of rare kidney diseases in policies and programs aimed at eliminating health disparities in communities of color. The HHS must also submit this update in the FY 2024 Congressional Budget Justification. Submitting these budget justifications ensures that the agencies are being held accountable for the funds they were given. Congress also requested that both the NIH and HHS provide updates in specific program improvements in the FY 2024 Congressional Budget Justification on disparities in kidney care and the inclusion of rare kidney diseases in policies and programs aimed at eliminating health disparities in communities of color. NephCure and its partners will be closely monitoring implementation of funding for these programs in the coming months. NephCure’s advocacy team is currently working with the new members of Congress and other organizations to prepare our policy priorities. Stay tuned for a robust grassroots effort engaging members in various activities, including our second annual Rare Kidney Disease Week this July. Once again, we thank our community for your commitment and support to helping advance legislative and policy priorities that would improve the lives of those living with rare, protein-spilling kidney disease. You can learn more and join the NephCure Action Network here. NephCure encourages its members to spend time getting to know their representative and senators.
FDA Grants FILSPARI Accelerated Approval for the Treatment of IgA Nephropathy in Adults February 17, 2023 by Kylie Karley Today marks an incredibly promising day for the rare kidney disease (RKD) community: the FDA has announced that the drug FILSPARI (sparsentan) has been granted accelerated approval for the treatment of IgA nephropathy in adults. The FDA’s accelerated approval program allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need. FILSPARI has been granted accelerated approval based on reduction of proteinuria. It is not yet known if the drug slows kidney function decline in IgAN patients. Over the next several months, Travere Therapeutics, the company that developed FILSPARI, will share more information about this new treatment and explain when and how those who need the drug can access it. “This is the moment we’ve all been working toward. FILSPARI’s FDA approval for IgA nephropathy is monumental for the entire rare kidney disease community. Today marks the start of a new era for the treatment of RKD — it is a testament to the research community’s and Travere’s commitment to our patients,” said Josh Tarnoff, NephCure’s CEO. “Our collective community is incredibly proud to reach this point, but we know the next phase of our vital work now begins: ensuring those who need this drug have access to it in time to save their kidneys. We’ve waited so many years for this treatment. Today’s FDA approval provides a beacon of hope that more needed new treatments are likely to be available soon.” The continued approval of FILSPARI may be dependent upon the data from Travere’s ongoing phase 3 PROTECT study. This study evaluates the drug in more than 400 patients with persistent proteinuria. After 36 weeks of treatment, patients receiving the drug achieved a nearly 50% reduction in proteinuria. While this is a new era for RKD patients and their families, we know that we still have a long way to go. FILSPARI is also being studied in FSGS patients, and it could be approved for the treatment of FSGS within the next year. With more than 60 clinical trial opportunities for RKD patients, even more new treatments are likely to be approved in the near future. We celebrate today, but we know we have not crossed the finish line yet. We must keep pushing research forward so we can continue to improve health outcomes for RKD patients. As Josh mentioned, we’re now facing a new challenge — ensuring all patients have timely and equitable access to these new and better treatments. We remain focused on our mission to empower people with rare, protein-spilling kidney disease to take charge of their health, while leading the revolution in research, new treatments, and care. We thank you for your incredible support that has helped us get to this day, and we are grateful for your continued dedication to our mission.