NephCure Kidney International Announces New Scientific Advisory Board Co-Chairs December 12, 2022 by Kylie Karley KING OF PRUSSIA, Pa. (Dec. 12, 2022) — NephCure Kidney International (NephCure) is excited to announce new co-chairs for the organization’s Scientific Advisory Board (SAB): Alessia Fornoni, M.D., Ph.D., and Matthias Kretzler, M.D. Alessia Fornoni, M.D., Ph.D. Drs. Fornoni and Kretzler, expert glomerular disease nephrologists who actively serve on NephCure’s SAB, will assume the roles of co-chairs following the term completion of former chairman Martin Pollak, M.D. “Dr. Fornoni and Dr. Kretzler have been indispensable partners of NephCure and the rare kidney disease research and care community, and we’re honored that they have agreed to lead our SAB,” said Josh Tarnoff, NephCure CEO. “Their combined ‘extensive research bench to bedside care’ experience and expertise have provided tangible progress and hope to the kidney community, an integral part of our mission. We are grateful for their passion and leadership to help push glomerular disease research and treatment forward.” Dr. Fornoni is a Professor of Medicine and Molecular and Cellular Pharmacology at the University of Miami Miller School of Medicine. She is the Chief of the Katz Family Division of Nephrology and Hypertension and serves as and Director and Chair of the Peggy and Harold Katz Drug Discovery Center. Through her pioneering work on insulin signaling, cholesterol metabolism and sphingolipid-related pathways, Dr. Fornoni uncovered novel pathogenetic mechanisms and therapeutic approaches for glomerular disorders. Dr. Kretzler is the Warner-Lambert/Parke-Davis Professor of Medicine/Nephrology and Computational Medicine/Bioinformatics at the University of Michigan. For the last 35 years of his life, he has focused his research efforts on studying glomerular failure and finding novel ways to treat it. Dr. Kretzler’s work has helped to redefined glomerular diseases in mechanistic terms. This framework is a driving force to develop and test novel precision medicine strategy for people with kidney disease, and hopefully bring the right drug to the right patient at the right time. Matthias Kretzler, M.D. Dr. Pollak, the previous chairman of the SAB, is a leader in the research of the genetic basis of rare kidney disease. His research lab discovered the genetic tie between people of African descent and focal segmental glomerulosclerosis (FSGS), a groundbreaking finding that has helped explain why Black Americans have a higher risk of kidney disease. Dr. Pollak continues working to identify genes involved in the development of FSGS in minority populations. Although he will be stepping down as chairman, Dr. Pollak will continue to sit on the SAB and serve the rare kidney disease community. Rare kidney disease is experiencing a revolution in research breakthroughs, care, and significantly expanded interest, which has led to NephCure’s responsibilities and programming significantly increasing. The addition of co-chairs reflect this important role to the SAB not only due to the responsibilities hosted in the position, but to help pave a way for new research in the kidney community. To receive more NephCure news and updates, sign up for our monthly newsletter here. About NephCure Kidney International NephCure Kidney International’s mission is to accelerate research for effective treatments for nephrotic syndrome, FSGS, and other forms of rare, protein-spilling kidney disease, and to provide education and support that will improve the lives of those affected by these conditions. 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 more than 60 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity.
Travere Therapeutics’ Sparsentan Drug Explained November 23, 2022 by Kylie Karley What is sparsentan and how is it taken? Sparsentan is a novel Dual Endothelin Angiotensin Receptor Antagonist (DEARA) being studied as a potential treatment option for immunoglobulin A (IgA) nephropathy and focal segmental glomerulosclerosis (FSGS). In clinical trials, sparsentan is being given as a tablet or age-appropriate liquid taken by mouth. What is the next step in the approval process? The U.S. Food and Drug Administration (FDA) has confirmed the Prescription Drug User Fee Act (PDUFA) target action date for sparsentan in IgA nephropathy is February 17, 2023. Can you describe the patients in the DUPLEX and PROTECT studies? DUPLEX and PROTECT are ongoing Phase 3 clinical trials studying the efficacy and safety of sparsentan. The DUPLEX study includes 371 patients, ages 8 to 75 years, with primary FSGS. The PROTECT study includes 404 patients, ages 18 years or older, with IgA nephropathy and persistent proteinuria despite angiotensin-converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy. Can you explain to our patients how sparsentan reduces proteinuria? The amount of protein found in the urine (called proteinuria) is seen as a marker of kidney function. Lowering proteinuria levels is associated with better kidney health outcomes. Pre-clinical data have shown that blocking both endothelin type A and angiotensin II type 1 pathways, in forms of rare chronic kidney disease, reduces proteinuria. In the PROTECT study, after 36 weeks of treatment, patients receiving sparsentan experienced a greater than threefold reduction of proteinuria from baseline (49.8 percent) compared to the active control irbesartan (15.1 percent). How is sparsentan different from other treatment options? Sparsentan is different from other medications because it is non-immunosuppressive and it is dual-acting (combines two mechanisms of action into one molecule). It selectively blocks the action of two important mediators of progression to kidney failure (endothelin type A and angiotensin II type 1) at their receptors. Can you give us an update about the use of sparsentan for FSGS patients? Travere Therapeutics anticipates having topline data from the DUPLEX study, including full two-year estimated glomerular filtration (eGFR) data, in the first half of 2023. The company is planning to pursue traditional approval of sparsentan for FSGS in the United States in 2023 and Conditional Marketing Authorization in Europe. What’s next for Travere Therapeutics? Travere Therapeutics is dedicated to working with the rare disease community to identify, develop, and deliver life-changing therapies. In addition to the development efforts for sparsentan in IgA nephropathy and FSGS, the company is advancing pegtibatinase for the treatment of classical homocystinuria, a genetic metabolic disorder that can cause life-threatening thrombotic events. In addition, early research efforts include partnering with leaders in patient advocacy and government research to identify potential therapeutics for Alagille syndrome, a rare genetic disorder that can affect the liver, heart, skeleton, eyes and kidneys. Where can patients with more questions go to find answers? For more information, patients should talk to their healthcare provider and can visit travere.com, rkdandme.com, navigateigan.com, or lowerproteinuria.com.
Travere Therapeutics, the IgA Nephropathy Foundation, and NephCure Kidney International Partner to Launch RKD & Me, a Campaign to Raise Awareness of Rare Kidney Disease November 15, 2022 by Kylie Karley Developed in collaboration with the rare kidney disease community, RKD & Me aims to amplify the experiences of people living with rare kidney disease. SAN DIEGO, Nov. 15, 2022 – Travere Therapeutics, Inc., the IgA Nephropathy Foundation, and NephCure Kidney International today announced the launch of RKD & Me, a new public awareness campaign that spotlights the real stories of people living with rare kidney disease to amplify their unique lived experiences, educate the public, and empower the newly diagnosed. With first-hand input from the rare kidney disease community gleaned from nationwide listening tours, the RKD & Me campaign further establishes the acronym, RKD, and provides a common space for those living with rare kidney disease to share their experiences on RKDandMe.com. In a world where millions of people live with chronic kidney disease, commonly known as CKD, it is estimated that only 60-80 per 100,000 people in the United States currently live with rare kidney disease (RKD). Due to low public awareness, people living with rare kidney disease can face challenges in getting diagnosed and finding support. “Rare kidney disease often presents with unique symptoms or no symptoms and can progress to end-stage kidney disease much faster than other forms of kidney disease,” said Kelly Helm, executive director of patient engagement at NephCure Kidney International. “This makes the experiences of people living with RKD substantially different from those with more common forms of CKD.” As part of the nationwide listening tour, the RKD community shared letters they penned to their younger selves recounting their experiences, offering words of advice and encouragement, and emphasizing the need for RKD to be delineated from other, more common kidney diseases. They also shared stories on how RKD intersects with every facet of their lives – including relationships, parenting, career and day-to-day activities. “By amplifying the personal letters and stories of the RKD community, the RKD & Me campaign will help people living with RKD learn from the experiences of others and find comfort in camaraderie as they navigate their journeys,” said Bonnie Schneider, director and co-founder of IgA Nephropathy Foundation. The RKD community’s letters are a focal point of this year’s launch and will continue to shape the multi-year campaign in the future. Anyone with an authentic connection to rare kidney disease is encouraged to submit a letter on RKDandMe.com to support raising awareness of RKD. “Through the listening tours and the letters, the RKD community shared about the realities of making space in their lives for rare kidney disease, and they also shared their strength, perseverance, and commitment to making their voices heard,” said Eric Dube, president and CEO of Travere Therapeutics. “Along with our partners NephCure and IgA Nephropathy Foundation, we hope the RKD & Me campaign will provide a much-needed platform that recognizes, celebrates, and elevates the unique experiences of people living with RKD.” To check out the powerful words of people living with RKD and submit your own letter, visit RKDandMe.com or follow #RKDandMe on social media. To learn more about rare kidney diseases and other ways to support people living with RKD, visit IgAN.org and NephCure.org. About Travere Therapeutics At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com. About IgA Nephropathy Foundation As the only dedicated patient advocacy group for people affected by IgA nephropathy, the IgA Nephropathy Foundation’s mission is to be a patient-centric organization focused on finding a cure for IgA nephropathy. Using the power of the patient community we are focused on funding research, using patient advocacy to empower our patients, and building a network of support. As a patient-run organization, we will work together with the hope of finding better treatment options and the ultimate cure. By patients, for patients. To learn more, visit www.IgAN.org. About NephCure Kidney International NephCure Kidney International’s mission is to accelerate research for effective treatments for nephrotic syndrome, FSGS, IgAN, and other rare, protein-spilling kidney diseases, and to provide education and support that will improve the lives of those affected by these conditions. 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 more than 50 interventional drug trials for primary glomerular kidney diseases. To learn more, visit www.NephCure.org.
NephCure Participates in Black Health Matters Summit and Health Fair: Rare Kidney Disease Across Generations Panel August 4, 2022 by Kylie Karley HOUSTON, TX (August 4, 2022) – NephCure Kidney International (NephCure) is hosting a panel discussion, “Men’s Health: Rare Kidney Disease Across Generations,” during the Black Health Matters Summit and Health Fair on August 6th in Houston, TX. Black Health Matters, a non-profit focused on improving the health and wellness of Black and BIPOC communities, hosts the largest public health forums on Black Health in the nation. This panel is sponsored by Travere Therapeutics. The “Men’s Health: Rare Kidney Disease Across Generations” panel includes three Black men, from the NephCure community, from a range of ages and in different stages of their focal segmental glomerulosclerosis (FSGS) journey; four time Grammy-award winning producer, songwriter, and social entrepreneur, Brian Kennedy, NephCure Board member and Kappa Alpha Psi fraternity member, Kevin Mott, and advocate and Georgia State University student, Joshua Albright. FSGS is a rare and progressive kidney disease that affects Black Americans at rates 4-5 times higher than white Americans. A mutation on the APOL1 gene, found in people of African descent, is associated with one of the most severe forms of FSGS. “These three NephCure advocates bring an essential voice and awareness to the Black community, especially amongst Black men. There is often a stigma when it comes to discussing men’s health issues. Brian, Kevin, and Joshua each have unique experiences that will shed light on and start a very important dialogue,” Lauren Eva, NephCure’s Vice President of Professional Relations, said. Each panelist will share their unique story with FSGS and discuss their experience pursuing an accurate diagnosis, navigating treatment options, and their mental health. Kennedy, Mott, and Albright will also ignite the critical conversation around race-related concerns, the genetic link that increases Black Americans’ risk for kidney disease, and the important early warning signs of kidney disease that everyone, no matter their age or health, should be aware of. The Black Health Matters Summit and Health Fair builds upon the collective strengths and shared goals of people and organizations seeking to disrupt the current medical establishment through preventative and intentional action that improves the quality of Black life. NephCure is proud to partner with Black Health Matters and Travere Therapeutics and raise awareness about rare, protein-spilling kidney diseases. The Black Health Matters Summit and Health Fair is both an in-person and hybrid event held on Saturday, August 6th. To learn more and to register for the event, please visit https://blackhealthmatters.com. About NephCure Kidney International NephCure Kidney International’s mission is to accelerate research for effective treatments for rare forms of Nephrotic Syndrome, and to provide education and support that will improve the lives of those affected by these protein-spilling kidney diseases. 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 more than 40 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity. About Black Health Matters Black Health Matters (BHM) was launched a decade ago, and is the leading health, wellness and chronic disease interactive digital platform dedicated to improving health outcomes among African Americans. BHM has collaborated with a wide range of partners, including fraternities, sororities, and faith-based and civic organizations to raise awareness around chronic diseases, mental and physical health, and fitness in the Black community. The organization’s mission is to improve health literacy, which includes addressing health care, health policy, health equity, and health disparities for positive outcomes. For more information go to www.blackhealthmatters.com ###
Delivering Cures in a Decade: NephCure Partners with PNRC to Provide Grant Opportunities for Pediatric Glomerular Disease Research July 13, 2022 by Kylie Karley KING OF PRUSSIA, PA (July 13, 2022) – NephCure Kidney International (NephCure), a nonprofit focused on finding better treatments for patients suffering from rare, protein-spilling kidney diseases, is proud to collaborate with the Pediatric Nephrology Renal Consortium (PNRC) to provide the Pediatric Glomerular Disease Accelerator Grant Program: Delivering Cures in a Decade. The Delivering Cures in a Decade grant program will provide small and medium-sized grants to nephrologists and researchers studying pediatric glomerular diseases, beginning this year, and running through 2024. This program aims to fund and accelerate research that will provide insights and evidence to help cure pediatric glomerular disorders. “The Pediatric Glomerular Disease Accelerator Grant Program is so exciting for our research community! Investigators in pediatric glomerular disorders are not short of innovative ideas and novel approaches to mechanisms of disease and potential therapeutics,” said John D. Mahan, MD, the PNRC Board President. “The gap that this grant program can really address is the current lack of critical seed funding to take these ideas from ‘concepts’ to ‘explored’, and as such lead to new treatments and cure. We could not be more excited or more committed to this work.” The NephCure and PNRC collaboration came from a mutual desire to promote and support areas of bench, translational, and clinical research in pediatric glomerular disease. “We are very hopeful that the NephCure and PNRC Delivering Cures in a Decade grant will provide invaluable insights and lead to changes of how we care for children affected by rare, protein-spilling kidney diseases,” Josh Tarnoff, NephCure CEO, said. This international grant program is open to any pediatric nephrologist or pediatric nephrology scientist. Applicants do not have to be a member of the PNRC to apply. The total award funds vary, from $10,000-$20,000 small awards to $50,000-$75,000 medium awards. Both NephCure and PNRC will review grant applications, and awardees will be announced in the fall at the PNRC members meetings. To learn more about the Delivering Cures in a Decade grant program application guidelines, eligibility, deadlines, and review process, visit https://pnrconsortium.org/nki-program/home. About NephCure Kidney International NephCure Kidney International’s mission is to accelerate research for effective treatments for rare forms of Nephrotic Syndrome, and to provide education and support that will improve the lives of those affected by these protein-spilling kidney diseases. 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 more than 40 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity. ###
Everything You Need to Know About TRACTION-2: Clinical Opportunity for FSGS and MCD Patients June 23, 2022 by Kylie Karley Goldfinch Bio is a biotechnology company based in Cambridge, MA which focuses on delivering disease-modifying precision medicines that bring hope and renewed quality of life to people living with kidney diseases. We recently spoke with Goldfinch Bio’s Chief Medical Officer, Ed Tucker, MD, who explains more about their Phase 2 clinical research trial for GFB-887, TRACTION-2, which is currently enrolling patients. Goldfinch Bio’s Chief Medical Officer, Ed Tucker, MD What patients are you hoping to enroll for this trial? In the TRACTION-2 study that is currently enrolling, we are looking for patients with focal segmental glomerulosclerosis (FSGS) and treatment resistant minimal change disease (TR-MCD) who are between the ages of 18 and 75 years old, have a UPCR greater than or equal to 1.0 g/g, and an eGFR greater than or equal to 30 mL/min/1.73 m2. Other inclusion and exclusion criteria will apply; please consult your healthcare provider for additional details. Why is FSGS and TR-MCD your target population? In the United States, there are currently no approved therapies for FSGS or MCD. Our treatment, GFB-887, was designed specifically for diseases like FSGS and TR-MCD because it targets and protects the podocytes that are damaged and lost in the disease development and progression. Other diseases involving podocyte injury/loss may be examined in the future as well. If someone enrolled in your Phase 2 study, what is the time commitment? After a patient has been evaluated (for up to 6 weeks) for eligibility in the study they will begin to receive the treatment and will continue for up to a total of 17 visits over approximately 26 weeks. Some of these visits are done via a phone call. After completion of the full study, patients will have the option to enroll in another “extension” study where they will continue to receive treatment for up to three years. An extension study is where all patients receive GFB-887 and allows for doctors to observe the effects of the therapy over a longer period of time. How many trial sites are there available? Where are these sites located? Currently, there are 76 locations throughout the United States where patients may participate in the study. Locations of these sites may be found here. What type of treatment is GFB-887? And what makes this treatment different from other ones being tested in clinical trials? GFB-887 is a tablet and is taken by mouth with water once a day. It differs from other medications in that it was created specifically to target and protect podocytes in the kidney to prevent damage and loss. No other therapies being tested for FSGS and TR-MCD work the same way that GFB-887 does on the kidneys. How is precision medicine integrated in this clinical trial? As this is a phase 2 trial, we are looking at different markers (including testing for genetic) for response to GFB-887, both before and after treatment has begun. Hopefully, we will be able to better integrate these findings into future studies and potentially improve outcomes for patients. Is there flexibility where lab samples can be collected for the participant? Is there an option for home care visits to collect these samples? Yes, patients have the flexibility to schedule follow-up visits within a few days before and after the expected date. Furthermore, if conditions outside the control of the patient change (Covid for example), or if the patient is unable to travel due to health limitations, the study site will work with the patient on scheduling and potentially offer telemedicine options for select visits. Additionally, your study site may be able to assist with travel services to and from office visits. What medicines can participants remain on while in the TRACTION-2 trial? Some medications, including calcineurin inhibiters (CNIs; cyclosporine for example), will need to be discontinued. Others, including angiotensin converting enzyme (ACE) inhibitors and angiotensin receptor blockers (ARB) should be continued, while various other drugs may be continued if on a stable dose. Please consult your healthcare provider for additional details. Does this trial have a placebo comparison? Yes. The placebo is used because comparing results in study participants who receive GFB-887 with results in participants who receive placebo is the best way to explore how well GFB-887 works and how safe it is. 66% of patients will receive GFB-887 (33% will receive placebo), but all patients will have the option to join an extension study where all patients will be offered GFB-887 for up to three years. What will happen to the results of this clinical trial? Can the participant stay on the medicine if it works? The results of this study will help guide the additional exploration of GFB-887 in these diseases and potentially others. It is one of many steps required before the FDA will approve a drug for broader availability. Currently, participants, once completing the initial study, have the option to participate in another “extension” study for up to three years. If a patient was taking placebo during the initial trial, they will receive GFB-887 in the “extension” study. Do you have plans for expanding into a pediatric trial? The current trial does not allow for patients under the age of 18, but we hope to offer additional options for younger patients in the future. Will the patients be paid for participating in the trial? Patients may be eligible to receive compensation for time and reasonable out-of-pocket expenses related to taking part in this trial. For more information, please talk to your trial doctor and trial staff. Goldfinch Bio’s TRACTION-2 clinical research trial is evaluating an investigational precision medicine, GFB-887, for the potential treatment of TRMCD and FSGS. The purpose of the trial is to determine if GFB-887 is safe and may help people who have high levels of protein in their urine due to kidney diseases caused by podocyte injury. To learn more about the TRACTION-2 clinical trial, click here. To see a full list of clinical research opportunities and find the right trial for you, visit KidneyHealthGateway.com. This article was developed in partnership with Goldfinch Bio, Inc.
NephCure Celebrates Introduction of New Era of Preventing End-Stage Kidney Disease Act April 15, 2022 by Kylie Karley KING OF PRUSSIA, Pa. (April 15, 2022) — NephCure Kidney International (NephCure) applauds Friday’s introduction of the New Era of Preventing End-Stage Kidney Disease Act (H.R. 7506) sponsored by Reps. G.K. Butterfield and Gus Bilirakis. This legislation would help transform the delivery of care to rare kidney disease patients by increasing community and healthcare provider awareness and education, addressing kidney health disparities in communities of color, and advancing rare kidney disease research. “I’m proud to introduce the New Era of Preventing End-Stage Kidney Disease Act, which is a critical step toward the transformation of kidney disease care,” said Butterfield, lead sponsor of the legislation. “For far too long, the rare kidney disease community has been overlooked, leaving many families in a chronic state of uncertainty as they navigate the many hurdles in getting diagnosed, treatment and care. There is a new era of hope on the horizon for these families.” “Our life-saving legislation will help remove diagnostic and treatment barriers for many patients suffering with a rare disease,” said Bilirakis. “Through increased education, 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.” Rare kidney diseases contribute to the more than $84 billion spent on treating Medicare beneficiaries with chronic kidney disease, including the $36 billion spent on treating people with end-stage kidney disease (ESKD), yet there has been little to no innovation in treatment for kidney disease patients since the 1960s when dialysis and immunosuppressants became commonplace. These treatment and diagnostic barriers, coupled with the lack of rare kidney disease awareness and education, often cause a delay in diagnosis that can result in a rapid decline in kidney function and, ultimately, kidney failure. For many rare kidney disease patients, their only options are dialysis, transplant or death. Additionally, communities of color are disproportionately affected by rare kidney diseases and face a lack of adequate treatment options due to existing health disparities — Black Americans are 4-5 times more likely to develop ESKD than white Americans. Many rare kidney disease patients struggle to find a nephrologist well-versed in their rare conditions who can provide an accurate diagnosis and expert care. FDA-approved treatments are lacking for most rare kidney diseases, but thanks to the 21st Century Cures Act we are on the cusp of a new era of rare kidney disease treatment and care — giving hope to thousands of patients and their families. By taking action now, Congress can promote health equity, save lives, and conserve valuable healthcare resources. “We applaud the Rare Disease Caucus Co-Chairs Mr. Butterfield and Mr. Bilirakis for their leadership. For decades there have been no breakthrough treatments for individuals experiencing rare kidney disease. However, we believe we are entering a ‘New Era of Kidney Care,’ and this comprehensive legislation will help forge a new frontier for innovation to thrive,” said Josh Tarnoff, NephCure CEO. “Dialysis and transplants cannot be the only options. It is time to modernize treatments and the delivery of care to kidney patients and bring it into the 21st century.” Revolutionizing rare kidney disease treatment has been a longstanding priority for NephCure. In 2020, a core group of partners, comprised of NephCure, Travere Therapeutics and the American Association of Kidney Patients (AAKP), facilitated the first-ever Rare Kidney Disease Roundtable and the resulting white paper entitled “We Deserve Better: Revolutionizing Rare Kidney Disease,” which outlined urgent needs for rare kidney disease patients and their families and helped inform the creation of the New Era of Preventing End-Stage Kidney Disease Act. NephCure will work with Congress to advance this bill as its key legislative priority. Patients and advocates are encouraged to join the NephCure Action Network, a movement of patients, caregivers, physicians and other partners who are committed to raising their voices to help educate policymakers on what’s important to those directly impacted by rare kidney diseases. For more information about the New Era of Preventing End-Stage Kidney Disease Act, visit NephCure.org/NewEraAct. About NephCure Kidney International NephCure Kidney International’s mission is to accelerate research for effective treatments for rare forms of Nephrotic Syndrome and to provide education and support that will improve the lives of those affected by these protein-spilling kidney diseases. 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 more than 30 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. exempt tax-exempt 501(c)(3) public charity. ###
NephCure Recognizes National Minority Health Month this April April 4, 2022 by Kylie Karley National Minority Health Month celebrates the inclusion of Asian and Pacific Islander, Black, Hispanic, Native American, and other communities of color in the conversation about health education and disease detection, disparities, and treatments. Chronic kidney disease (CKD) affects 1 in 7 adults in the U.S., however, for people of color, a combination of systemic racism, lack of inclusion in medical advancements, and other uncontrollable risk factors contribute to even higher CKD rates. CKD in Communities of Color If you are Black or African American, Hispanic or Latino, Asian American, Pacific Islander, American Indian, or Alaska Native, or Native Hawaiian or Other Pacific Islander, heritage you may be at an increased risk for kidney disease. Minority populations are also at higher rates of high blood pressure, diabetes, obesity and heart disease — all of these factors increase the risk for kidney disease. Did You Know? Asian Americans and Native Hawaiians are the most understudied racial group in CKD research. Black Americans represent 13% of the population, but account for 32% of CKD patients. Hispanic Americans are 1.3 times more likely to develop kidney disease, and CKD rates have risen 70% since 2000. Our Health Equity Initiative With the inequities in kidney health in mind, NephCure launched a Health Equity Initiative in 2021 to address racial disparities in rare kidney diseases. NephCure acknowledges the voices of these communities, and we are excited to continue the work of addressing health disparities for all multicultural communities by featuring culturally relevant health education and resources, partnering with community-based organizations, and advocating for diverse and just medical research.
The Difference a Doctor Makes: Christine’s Fight for Daughter’s Health December 8, 2021 by Kylie Karley Christine Floto and her daughter, Madi Madi was only 4 years old when she faced potential kidney failure. It took Madi’s mom Christine years to realize these harsh medications were not only failing to improve Madi’s kidneys but were making her sicker overall. Madi’s nephrologist insisted the current medications were the best they would get. Christine asked for further testing, but they denied her requests as Madi’s kidneys worsened. Then Christine found NephCure. She attended a patient summit and broke down in tears when she learned there were better options for her daughter. Madi no longer had to suffer. Generous support from people like you helped NephCure connect Christine with an expert doctor who listened to her concerns. He ordered a genetic test, revealing that Madi has an extremely rare genetic cause of focal segmental glomerulosclerosis (FSGS). Madi was enrolled in a clinical trial, which gave her access to cutting-edge medication. With the help of NephCure and her new doctor, Madi got her life back. Her kidney disease is slowing, she gained 30 pounds, and is doing better than she has in years! “Through NephCure, I learned more about Madi’s disease and gained the confidence to seek care from top specialists,” Christine said. We have entered a new, unprecedented era of rare kidney disease care. Now more than ever, NephCure is laser-focused on ensuring all patients have access to the latest research and care. NephCure offers online education and support programs that empower rare kidney disease patients and their families, just like Madi and Christine. Through our NephCure Specialists program, we help connect patients with expert doctors who provide the latest treatment options. We are expanding local communities to help connect people to peer support, but more work is needed. “We will forever be grateful for NephCure’s education and support,” Christine said. “It’s given our family our life back.” Will you donate to help us ensure that every patient receives the best care and support possible? To make a contribution to our organization before the year end, please click here. Thank you in advance for your generosity.
NBA Hall-of-Famer Alonzo Mourning Shares Personal Story to Raise Kidney Disease Awareness November 18, 2021 by Kylie Karley KING OF PRUSSIA (Nov. 18, 2021) – NBA hall-of-famer Alonzo Mourning was at the height of his basketball career—he had just won gold in the 2000 Summer Olympics when he noticed extreme swelling throughout his body and a lack of energy. A routine physical exam showed abnormalities, and eventually, Mourning received a diagnosis of a rare, protein-spilling kidney disease called focal segmental glomerulosclerosis (FSGS). “I said ‘Doc, am I going to die?’ He paused; he took too long to answer me. And he said, ‘We have no known cure’… ultimately, he said, ‘In about 10-12 months you’ll probably be on dialysis,’” Mourning said as he recalled the jarring day. Mourning, who missed the entire 2002-2003 season due to kidney disease and later went on to win the World Championship with the Miami Heat in 2006, publicly recounted his personal journey with FSGS in a video for NephCure Kidney International’s kidney disease awareness campaign, debuting Nov. 18. In highlighting Mourning’s story, NephCure aims to reach those who are at risk for kidney disease and to educate the public about the signs and symptoms of these conditions. Black Americans are 4-5 times more likely to develop kidney failure than white Americans, and 1 in 8 are at risk for a genetic form of kidney disease. A variation on the APOL1 gene contributes to this disparity. “We are proud to work with Alonzo Mourning in sharing such an important message that will inspire and inform others who fall within this at-risk population. Our goal is to find a cure for this debilitating kidney disease, as well as educate and support those who are affected by it,” said Michael Levine, NephCure Board President and kidney disease patient parent. This awareness campaign comes just months after NephCure launched its Health Equity Initiative, with the goal of ensuring equitable access to advancements in research, treatments, and care, and to reach individuals from communities of color earlier in their disease progression, preventing or delaying their need for dialysis and transplantation. Support for this video and the awareness campaign was provided by Vertex Pharmaceuticals, Travere Therapeutics, and other funders of the Health Equity Initiative. To watch the entire video with Alonzo Mourning, click here. About NephCure Kidney International NephCure Kidney International’s mission is to accelerate research for effective treatments for rare forms of Nephrotic Syndrome, and to provide education and support that will improve the lives of those affected by these protein-spilling kidney diseases. 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 more than 30 interventional drug trials for primary glomerular kidney diseases. NephCure is a U.S. tax exempt 501(c)(3) public charity. Learn more at NephCure.org. ###