NephCure

PARASOL, an initiative co-sponsored by NephCure, the International Society of Glomerular Disease, the U.S. Food and Drug Administration, the Kidney Health Initiative, and the National Kidney Foundation, convened for the third time in the past ten months at a public scientific workshop held in Bethesda, Maryland on October 7-8, 2024. At the meeting, the biostatistical team reported an in-depth analysis of 1626 children and adults with FSGS from a combined dataset drawn from multiple glomerular disease networks around the world. Analysis lead Abigail Smith, PhD, Associate Professor of Biostatistics, Department of Preventive Medicine, Northwestern University Feinberg School of Medicine, reported: “The principal finding is that reduction in proteinuria over 24 months is strongly associated with a reduction in the risk of kidney failure, and responder definitions based on thresholds of proteinuria are both biologically plausible and strongly supported by epidemiological data. The finding was consistent in subgroups based on patient age, level of baseline proteinuria, or presence of chronic kidney disease and was validated in a large independent European patient cohort.” Discussion of the findings in an open forum highlighted their broad utility, the biological role of proteinuria in FSGS as a podocytopathy, and implications for clinical trial design. The results of PARASOL will be presented to the full nephrology community on October 25, 2024 at a dedicated session of the annual Kidney Week meeting of the American Society of Nephrology in San Diego, California. Plans for publication in a peer-reviewed journal are also underway. To read the full press release, visit here: https://lnkd.in/gY8DTFX8

We’re thrilled to highlight Rachel Slattery, our Associate Director of Development for her contributions and 5 years of driven passion. Her hard work has brought way to many successful events and fundraisers. Dive into her Q&A below! What has been your favorite project at NephCure that you have overseen? "My favorite accomplishment at NephCure has always been overseeing NephCure’s signature fundraising gala, Countdown to a Cure NYC. It’s truly special to witness the event come together—seeing our incredible community of donors, sponsors, researcher, patients, and families unite to push our mission forward!! With the help of our amazing Chair, Michael Levine, plus NephCure Board Members & Team, we’ve been able to grow this event year after year, raising crucial funds and awareness for RKD. It’s truly humbling to be part of something that has such a direct and lasting impact on people's lives." How long have you been with NephCure? "I’ve been with NephCure for just over 5 years now, and every moment has been a rewarding experience. It's been a privilege to watch the organization evolve and be part of something so meaningful from the inside out." What excites you about working with NephCure, or what motivates you the most? "What excites me the most is the knowledge that the work we do truly matters. Every day, we get closer to making a real difference for people affected by RKD. Hearing stories from patient families and knowing that our efforts are helping to bring hope keeps me going. It's truly inspiring to be part of something that touches so many lives in such a impactful way!"

The PARASOL Project meets again — this time in Washington, DC — to advance the understanding and use of proteinuria and eGFR-based endpoints as surrogate endpoints for accelerated and traditional approval in FSGS. PARASOL stands for 'Proteinuria and GFR as Clinical Trial Endpoints in Focal Segmental Glomerulosclerosis,' and the goal of the meeting is to make sure there is an endpoint for FSGS clinical trials. An endpoint is a measurable outcome in a clinical trial that helps determine the safety and efficacy of a treatment. “When this project was initially scoped, it was about two and a half years worth of work, and that was clearly not going to be what we need. So the community came together and we got this down to 11 months! It took an incredible amount of work,” NephCure CEO Josh Tarnoff said. This global initiative looks at the data from 28 databases across the world. This scale of data sharing and analysis is largely because the need is so great— there are currently no approved therapies for FSGS. “The goal is to give FDA confidence in the modeling to generate reliable and reassuring data, give sponsors incentives and motivation to continue FSGSS investment knowing there will be a feasible and reasonable regulatory path ahead…and most importantly, give patients the hope that studies will achieve new drug approvals,” NephCure Board Member and PARASOL meeting attendee, Barbara Gillespie, MD, FASN said. Today more than 80 researchers, biostatisticians, industry partners, government regulators, patient advocates, and nonprofit organizations are attending in person; with roughly 80 more attendees joining virtually. “I’m so proud of nephrology, we’ve all stepped up! We can accomplish so much together,” PARASOL Project Co-Chair, Dr. Laura Mariani said. NephCure is proudly partnering with the International Society of Glomerular Disease, National Kidney Foundation, Kidney Health Initiative, and the U.S. Food and Drug Administration (FDA) to sponsor the PARASOL Project. The PARASOL Project meeting continues tomorrow, October 8, 2024, followed by broader dissemination at American Society of Nephrology Kidney Week later this month in San Diego. 

Meet Ariana Barton, a passionate advocate for the rare kidney disease community and a valued member of the NephCure family. Ariana's dedication shines through in everything she does, and we're proud to share her experience as a volunteer in her own words. "I learned about NephCure through social media just a few months into my IgAN journey. One of my friends with IgAN shared one of NephCure’s posts and that led me to the website where I found out about a Rare Kidneys Connect event that was happening near me. I signed up to attend, and that was one of the best decisions I’ve made. My mom attended with me and we both felt it was informative and empowering. I left with a better understanding of my diagnosis and the tools that were available to me as a patient. I’ve also made friends through NephCure, and I think that has shaped my journey more than anything. Having a community of people who understand without judgment has been a huge advantage for me, especially in regard to my mental health." "I knew I wanted to volunteer for NephCure as soon as I connected with Kimberly Figgs. She is so welcoming and easy to talk to, and has made volunteering an enjoyable and smooth process. It has been so much fun helping at NephCure events and I love that I can volunteer on my own terms."   "My favorite memory with NephCure so far was my first patient summit, last year in San Diego. Though it wasn’t my first time meeting other RKD patients, I loved that it was spread over the entire weekend to give us plenty of time to meet new people, rest, and enjoy our time outside of learning and soaking up valuable information. It was a great experience, and I came home so much more informed and ready to have some new discussions with my nephrologist." "I would encourage anyone who is on the fence about becoming a part of the NephCure community to join us! This is one of the friendliest and most welcoming group of people I’ve ever had the pleasure of working with. I have nothing but great things to say about NephCure!" Thank you, Ariana, for your commitment and for helping make a difference in the lives of those affected by rare kidney diseases! Want to make an impact? Learn how to get involved at NephCure.org.

We're so honored to have been present alongside the International Society of Glomerular Disease at the #ESPN2024 conference in Valencia, Spain! This conference was an incredible opportunity to explore cutting-edge advances in pediatric nephrology. Our CEO, Joshua Tarnoff, had the privilege of attending with esteemed colleagues Laurel Damashek, Dr. William E. Smoyer, M.D., and Dr. Gema Ariceta. A heartfelt thank you to the European Society of Paediatric Nephrology for organizing such an insightful and impactful event.

Today is an important day for our clients at NephCure who are recognizing IgAN Awareness Day. IgA Nephropathy is a rare kidney disease that causes inflammation in the kidney's filtering units, potentially resulting in kidney damage or kidney failure. To help raise awareness of this "silent" kidney disease in support of NephCure's mission, we're wearing our shirts in solidarity! Emma J. S. Magnus, LSW

Did you know that up to 40% of people living with IgAN show no recognizable symptoms at initial diagnosis? Today is IgAN Awareness Day, and we're working to raise awareness and empower patients with this often 'silent' but serious rare kidney disease. IgAN often lurks silently in its early stages, potentially going undetected for years due to a lack of symptoms. This is why we're working to raise awareness and provide resources and education to patients and caregivers, who are facing the challenges of this disease. Join us in raising awareness and show your support by reposting, and learning more information. A huge thank you to the generous support of our IgAN Empowerment sponsors, Travere Therapeutics, Calliditas Therapeutics, Alexion Pharmaceuticals, Inc., and Otsuka Pharmaceutical Companies (U.S.). https://lnkd.in/gWTd-ikY

Today, we’re proud to sponsor NephCure's first-ever IgAN Awareness Day. This milestone day aims to spread awareness about #IgANephropathy and all those affected by this silent but serious #RareKidneyDisease. Learn more about #IgANAwarenessDay2024: https://lnkd.in/e4id9MhW

We continue to mark C3G Awareness Day 🗓️ Rare kidney diseases, including C3G and IC-MPGN, create significant uncertainty for those affected 💛 National kidney health plans could support optimal care and better outcomes for all afftect patients and their families 👨👩👦. 🙏 European Reference Network, ERKNet, International Society of Nephrology, EKHA - European Kidney Health Alliance, European Renal Association (ERA)

Founded in 2000 by a group of committed patient parents, NephCure is the only kidney organization squarely focused on nephrotic syndrome, FSGS, IgAN, and other rare, protein-spilling kidney diseases. During this time, there was little to no awareness for rare, protein-spilling kidney disease. Since our founding, NephCure has invested more than $40 million in kidney disease research. We’ve helped create a new reality where patients have access to new treatments, including more than 60 interventional drug trials for rare kidney disease currently in progress.