NephCure Applauds the Introduction of Federal Legislation to Improve Kidney Patient Access to Early Interventions, Improved Diagnostics & Access to Treatments

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.

Q&A with Dr. Kopp of the NIH

picture- KidneyWordCloudDr. Jeffrey Kopp is a physician and researcher who focuses on FSGS and related diseases. He currently leads a group in the kidney disease section (officially called the National Institute of Diabetes and Digestive and Kidney Diseases, or NIDDK) of the National Institutes of Health (NIH). Dr. Kopp is also working on a new clinical trial for FSGS, MCD, and MN patients at the NIH headquarters near Washington D.C. We had the awesome pleasure of sitting down and catching up with Dr. Kopp about his fascinating job and new clinical trial. Keep reading to learn more, and read about some of his other research projects here. 

Interview highlights:

  • Dr. Kopp works at the National Institute of Health’s kidney branch, where he studies glomerular diseases such as FSGS and MCD. He also serves as Captain for the United States Public Health Service, and has been deployed to help with medical care during natural disasters.
  • Dr. Kopp is leading a new clinical trial for FSGS, MCD, and MN patients at the NIH studying a compound called ManNAc as a treatment option. 
  • ManNAc is a sugar that occurs naturally in your body. Another researcher at the NIH found that mice without ManNAc developed MCD, and adding ManNAc to their diet was helpful in treating it. Therefore, it may be effective at treating MCD, FSGS, and MN in humans (Dr. Kopp describes the full mechanism below—make sure you read the article!)
  • This study requires people to stay at the NIH for 11 days total, but it can be split up into 2 trips. Luckily, there is a lot to do to pass free time you may have at the NIH, including movie marathons, exercise programs, an art gallery, and an in-house business center.
  • Learn  more about taking part in the study by clicking here or contacting Emily Brede, RN at

Full interview:

NKI: What is your job at the NIDDK?

Jeffrey B. Kopp, M.D.
Jeffrey B. Kopp, M.D.

Dr. Kopp: I am fortunate to lead a translational research group at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), which is part of the National Institutes of Health. Our mission is to develop a better understanding of the disease mechanisms responsible for focal segmental glomerulosclerosis (FSGS) and to develop more effective and less toxic therapies.

I also serve in the United States Public Health Service, with a rank of Captain. My primary mission at NIH is to carry out basic and clinical research in FSGS. I also deploy for public health emergencies, such as natural disasters. Thus, I participated in the medical response to Hurricanes Katrina and Ike.


Dr. Kopp: The NIH is a federal biomedical research facility located in Bethesda, MD. The campus includes a 240-bed Clinical Research Center and extensive outpatient clinics. Every patient who comes to NIH participates in a research protocol. Some protocols involve novel treatments and other protocols involve giving samples for research. NIH physicians may give advice about standard therapies that can be used. There are no charges for any medical care provided by the NIH Clinical Center.

NKI: What do you enjoy about CKD research?

Dr. Kopp: CKD, and particularly glomerular diseases (such as FSGS), are incompletely understood, and the available therapies are not ideal. I like the challenge of understanding and treating these diseases, and most of all I like the opportunity to improve the lives of patients with these conditions.

NKI: The newest clinical trial for FSGS, MCD, and MN patients at the NIH is looking at MaNAc as a treatment option. Why did you decide to study MaNAc?

Dr. Kopp: A colleague at NIH developed mice unable to make ManNac. She found that these mice developed glomerular disease soon after birth. This disease resembled a human glomerular disease, minimal change disease. Providing extra ManNAc orally to the mice cured the kidney disease. This prompted the question: can we use ManNAc to induce remissions in our patients?

Chemical Structure of ManNAc

NKI: What is ManNAc?

Dr. Kopp: Perhaps the word sounds to you like manna, the food the Israelites found in the desert and that helped sustain them. There is a tree in Europe that exudes a sweet white resin, similar to the sap of the sugar maple, and people who knew the Bible story called the tree the manna tree. A chemist found a distinctive and novel sugar in the manna resin, and he called the new sugar “mannose”.

NKI: Does ManNAc occur naturally in the body? Is it found in food?

Dr. Kopp: ManNAc is a natural product and essential for good health. Our food does not contain much ManNAc. Our bodies make ManNAc, which is converted in our cells to mannose. This in turn is converted to sialic acid, which is put on many proteins. All of these are sugars, but they differ from glucose in that they are not related to diabetes and they are present in very small amounts, so that they do not add calories in the diet.

NKI: What is the reason for believing that ManNAc might be useful in treating glomerular diseases?

glovesDr. Kopp: Podocytes are cells on the outside of the kidney glomeruli and serve to prevent plasma proteins from leaking into the urinary space. Many patients with glomerular diseases have lost sialic acid from the proteins on the podocyte. We think that providing extra ManNAc might promote the return of sialic acid to podocyte proteins and that this might improve podocyte function. We see some evidence in mouse models of FSGS that supplemental ManNAc in the diet helps treat these mice.

NKI: What is involved for patients in this study?

Dr. Kopp: Patients will provide their medical records for review by the NIDDK team. We also review the kidney biopsy materials from past kidney biopsy. No kidney biopsy is done as part of this study. If patients appear to qualify for the study, they will come to NIH for an outpatient visit for evaluation and to discuss study participation.

NKI: Is travel to NIH paid for?

Dr. Kopp: Travel to NIH can be arranged and provided by NIH. If overnight accommodation is needed, NIH can provide this also.

NKI: Why are patients required to stay at the NIH during this study?

NIH Headquarters
NIH Headquarters

Dr. Kopp: The study requires being an inpatient for 11 days, either as a single stay or as two stays of five and six days. The reason for the inpatient stay is allow frequent sampling of blood and urine and for safety, to be sure there are no side effects.
NKI: What can patients do with any “free time” during the study? How much free time do you expect patients to have?

Dr. Kopp: During the first five days, there are frequent time points for sample collection. During the second six days, samples are needed at 8 am and 8 pm. There is extensive free time that patients can use as they like.

There are many activities that can help pass the time at NIH

• Patient Computers combination television and computer (with Internet access) at most patients’ bedsides to provide access to games, web browsing, and personal e-mail via the Internet

• Patient Library has more than more than 5,000 books, including a selection of current best-sellers, reference, foreign language, large-print, picture, and audio books

• Clinical Center’s Fine Art Program has more than 2,000 works of art. Most artwork remains on permanent display throughout the hospital, but there are six galleries on the first floor that change every eight weeks. A walking tour is available to assist patients, caregivers and visitors in their enjoyment of the artwork on display.

•Recreation Therapy programs include:
o Arts and crafts
o Music
o Games and sports
o Social events
o Exercise
o A large selection of DVD movies
o Instruction in coping skills such as relaxation, enhanced communication, and stress management

• Spiritual Care Department offers Catholic, Jewish, Islamic, and Protestant services in the interfaith chapel

• Business Center has four PCs and four MACs (all with Internet connection) as well as a combined printer/copier/FAX and telephones are available.

NKI: Who can participate in the ManNAc study?

Dr. Kopp: We are recruiting adults (age ≥18 years) with a primary glomerular disease, including minimal change disease, FSGS, and membranous nephropathy, and with nephrotic range proteinuria (urine protein/creatinine ratio > 2 g/g).

Exclusion criteria include having diabetes mellitus and receiving pulse therapies, such as rituximab. Monetary compensation is provided.

NKI: How do I get more information about the study?

Dr. Kopp: The study, like all clinical research studies, is described at
You also contact the study research nurse, Emily Brede, RN at

The Unique Adult Experience Webinar

“As far as I knew, kidney disease didn’t run in my family. Most important, I didn’t have time for kidney or any other kind of disease.”dine

Dine Watson, author of the Washington Post article “Kidney disease? I was only 33 years old, and I felt fine” will be featured on our next webinar, The Unique Adult Experience. Diagnosed with FSGS in 1984, Dine’s story will resonate with all adults living with Nephrotic Syndrome and the related diseases.

During this webinar, Dine will offer insight about all aspects of living as an adult with kidney disease, and also how to hope and how to find a community.

Register now to reserve your spot!

Hope to see you there!

Questions? Contact Chelsey at 610-540-0186 ext. 19 or

2015 Countdown to a Cure – THANK YOU!

We’ve said “hello” and “goodbye” to another Countdown to a Cure…

Bravo Events - 2015From the beautiful scenery of New York City’s Chelsea Piers, to the heartfelt speeches delivered by NephCure’s beloved family, the Jones’ and honoree, Olympic athlete Aries Merritt, this event was truly the “Chance of a Lifetime” to make a difference in the fight against Nephrotic Syndrome. The success of Countdown has always been measured by the generosity of the many hundreds in attendance who consistently show support for NephCure and this year, our expectations were overwhelmingly exceeded, for which we could not be more grateful. You are changing the story.

Red Carpet, Nephcure-6

Energies were high and attendees were excited, lighting the way for one of the most successful galas in NephCure history. “Fund a Cure” donations blew expectations out of the water, bidding was at a high and the second annual game of “heads or tails,” brought a touch of silliness to an evening surrounding a very serious cause. Emcee, Moody McCarthy, along with “Asbury Fever,” a Bruce Springsteen Tribute band, kept the party going all night long with many moments filled with laughter, dancing and mingling.


As always, we want to thank the committee who worked so hard to put this event together and the volunteers who generously gave up their time to help this event flourish into a huge success. The 2015 New York Countdown to a Cure raised over $750,000 and many left the event feeling inspired and hopeful.

Finally, thanks to YOU. To each of you reading this who’ve decided to join us in this fight.

We can’t do this alone.
We need you, we’re grateful for you and we thank you.

BRAVO EVENTS -- "NEphcure Event" -- Pictured: (l-r) -- (Photo by: Heidi Gutman/Bravo)

A Call for More Specific Standards of Care

From a new article, published by Nephrology News and Issues, comes a provocative call for more specific standards of care in nephrology. As of now, the standard of care for the majority of kidney-failure patients is to follow a similar treatment plan, regardless of the cause for their kidney failure. Yet, according to research conducted at Stanford University School of Medicine, this approach is not enough. In fact—it could even be dangerous.

Researchers used data from over 84,000 patients, who between 1996 and 2011, suffered end-stage kidney disease due to one of six major glomerular disease types. The results were shocking. Mortality ranged all the way from 4% per year for patients with subtype, IgA nephropathy, to 16% per year for patients with subtype, vasculitis. Furthermore, patients with lupus nephritis were almost 2x as likely to die as those with IgA nephropathy. In other words, the specific type of glomerular disease determined how long a patient lived after developing kidney failure. As one researcher put it, “when you divide patients according to their glomerular disease subtype, you actually see a whole spectrum of outcomes (O’Shaughnessy).” And yet, the current standard of care is to follow a similar treatment plan for most kidney-failure patients, regardless of cause.

“We showed that a patient’s cause of kidney failure is strongly associated with their risk of dying after starting dialysis or receiving a kidney transplant.” Thus, treatment can no longer be generalized and non-specific. Medical professionals cannot ignore the cause of kidney failure and proceed with treatment as though all kidney failures are one and the same. The cause of kidney failure cannot be forgotten. Rather, it should be the stepping point from which treatments are determined, and tailored toward disease-specific risks. More so, further research is necessary to determine why these survival disparities exit from one patient to the next. If medical professionals begin to take into consideration what caused the kidneys to fail in the first place, it could possibly improve the patient’s quality of life and even increase their life span following kidney-failure.

To read more, visit:



The Levine family just took great steps to STAND UP & BE COUNTED. Meeting with the Congressman, they took just another step for the best representation and advocation for those with Nephrotic Syndrome and FSGS. Sydney Levine, who recently started a viral social media campaign with the #SUBCselfie project, caught the attention of the Congressman as well as many famous, powerful voices across the country. As a result of the Levine family, who never stop trying to find ways to beat FSGS for son and brother, Matthew, the right people are becoming aware and joining in the fight. The Congressman will be taking this to appropriations on the hill next week!

Why YOU Should Attend a Community Cafe

We know that spring can be extremely busy for everybody, but we wanted to share with you the top 10 reasons why YOU and your family should attend a local Community Cafe Patient Workshop! 
REGISTER for one in your area! 


10.     Access to leading experts in the Nephrology world

9.         Enjoy kidney friendly, low sodium food

8.         Learn what 3 questions to ask your nephrologist at your next appointment

7.         Find out what events are happening in your area

6.         Get the inside scoop on research

5.         Meet others and stop feeling alone

4.         Hear the do’s and don’ts from a renal dietitian

3.       Empower yourself with scientific knowledge

2.      Become part of a global community working towards a cure

1.      You owe it to yourself to know everything about what’s happening


Ann Arbor- May 14 

Check out our Peer to Peer Programs!

imagesOne of the most important things we do at NephCure is connect people to others experiencing similar challenges of living with chronic kidney disease. Whether it be understanding the complexity of Nephrotic Syndrome/FSGS, the side effects of medications, frequent trips to the nephrologist or navigating your way through labs and diets, it can be incredibly overwhelming. At NephCure, we understand the value of connecting patients and caregivers with others to share experiences, frustrations and, better yet, good news!

That’s why we’re expanding our Peer to Peer Support Program. In addition to our online support community, NephSpace, we offer our Patient to Patient Connections (P2PC) program. P2PC is a worldwide organized network of patients and caretakers whose lives have been affected by the diseases causing Nephrotic Syndrome and FSGS.  It is designed to connect individuals via email or phone based upon any or all of the following: diagnosis, symptoms, and complications, age of individuals or sometimes specific geographical area.  Our volunteer patient/caretaker ambassadors are committed to offering support and sharing their experiences with others who are facing similar challenges.  You can learn more or be connected with a patient/caretaker ambassador here

We encourage you to take time to make connections with others in similar situations as we know you will benefit greatly from the support and sharing

 If you are interested in becoming Volunteer Patient/Caretaker Ambassador please visit or contact Kelly Helm at

Seeking Volunteers for NEW Clinical Trials Ambassador Program

Have you ever participated in a clinical research study? Are you willing to share your experience with others? If so, then you would make a fantastic addition to our newest initiative to find better treatments and a cure for Nephrotic Syndrome: the Clinical Trials Ambassador Program! NKI is seeking volunteers from across the country to act as Ambassadors to other patients and the medical community. Ambassadors will spread knowledge and information about the importance of clinical research in the search for better treatment options and a cure.

Ambassadors can expect to spend between 5 and 10 hours a month volunteering in various ways that will help spread awareness and knowledge about clinical research. If you are interested in learning more about the Clinical Trials Ambassador Program, contact Chelsey Fix at

NephCure Kidney International Announces NKN Steering Committee Co-Chairs

We are pleased to share that Kathleen Broderick and Randall Snyder were recommended and have accepted their nominations as Steering Committee Co-Chairs for the NephCure Kidney Network (NKN). Kathleen was considered a strong candidate due to her dual experience as a patient caregiver as well as a patient advocacy representative. Similarly, Randy’s dual perspective as a patient caregiver and clinical provider give him unique insight relevant to the NKN.

Kathleen is a member of the NephCure Board of Directors and Research Committee and an editor at DeGruyter Publishing, a Science, Technology, and Medicine (STM) publisher of professional books and scholarly journals in Boston, Massachusetts. Additionally, Kathleen cares for her 16-year-old son, who was diagnosed with Nephrotic Syndrome at 20 months.

Randy is an interventional radiologist with his master’s in molecular biology living in Medford, New Jersey, who cares for his son who was diagnosed with focal segmental glomerulosclerosis.

Kathleen and Randy will be facilitating Steering Committee calls and communication moving forward. Please join us in welcoming Kathleen and Randy as the inaugural NKN Steering Committee Co-Chairs!

Claritas Genomics Launches Convenient Genetic Testing Option For Nephrotic Syndrome Patients

On September 11, 2014, Claritas Genomics in Cambridge, MA, announced the creation of its newest genetic screening panel, which tests gene variants commonly associated with steroid resistant Nephrotic Syndrome in children. The test uses next-generation gene sequencing techniques to detect mutations in the 28 most common gene variants associated with Nephrotic Syndrome, making it the largest genetic panel for any pediatric kidney disease.

Typical candidates for this genetic test include: 1) pediatric patients less than 12 months old; 2) pediatric patients that do not respond to steroid therapy; 3) patients with a family history of Nephrotic Syndrome; 4) pediatric patients with features of FSGS; and 5) patients who have congenital malformations associated with Nephrotic Syndrome.

In a time where healthcare is becoming more patient centric, as opposed to disease centric, genetic screening may lead to more ‘personalized’ treatment based on the characteristics of a patient’s disease profile. Information about genetic underpinnings of Nephrotic Syndrome may inform what drugs are used for initial treatment, provide improved information for patients and families about the likely clinical course of disease, could improve the selection of appropriate transplant donors and provide opportunities for genetic counseling for other family members.image003

“This is the start of an exciting new era in understanding genetic factors that play a role in the onset and progression of at least some cases of Nephrotic Syndrome. NephCure Kidney International is proud to have supported the research at Boston Children’s Hospital that led to the development of this commercial application,” says Marilyn Hailperin, National Director of Research for NephCure. “Clear guidelines for when to seek genetic testing and the impact on patient treatment still need to be developed, but the availability of tests such as ClariFocus will help to advance scientific knowledge that will lead to better therapies.”

Patients and their family members should talk with their nephrologist about this new test. It is only available with a physician order at this time.

Jet Food Stores annual NephCure Golf Classic set for May 6, NASCAR Driver David Ragan and Former UGA Football Phenom David Greene to Attend

May 1, 2014

Jet-Food-Store-LogoOn May 6, Jet Food Stores will host the NephCure Golf Classic at Twin City Country Club in Sandersville, Georgia. The event will benefit the NephCure International Kidney Foundation, an organization committed to supporting research seeking a  cure for the potentially debilitating kidney disease Focal Segmental Glomerulosclerosis (FSGS) and Nephrotic Syndrome.

This is the 15th year of the golf tournament and the 11th it will benefit NephCure. The event has generated more than $350,000 for the NephCure mission since its inception. Forty-five foursomes have registered for the event. 

TurnerFamilyNephCure board member and Jet Foods President Charles Turner is the driving force behind the event. This cause is important to his family and their goal of supporting research to discover and make available better treatments for all families dealing with these kidney conditions.

NASCAR driver and NephCure Ambassador David Ragan will attend. The Ragan family has a long history with Jet Food Stores. “We sponsored Ken Ragan 30 years ago,” said David Usry, vice president at Jet Food Stores. “We then started sponsoring his son David when he was 10 years old. We’ve sponsored him throughout his career. We’ve always had a great relationship with them; a friendship more than anything. [David] is a great guy and he has never forgotten where he came from,” Usry concluded. Ragan drives the No. 34 Ford for Front Row Motorsports in the NASCAR Sprint Cup Series.

Another standout attendee will be David Greene, a former quarterback from University of Georgia. “He’s like Joe Namath around here,” Usry said. “He’s a great guy with a lot of Character.” Greene played for Georgia from 2000 to 2004. During his tenure, he set the NCAA Division I record for wins (42), a record set previously by Peyton Manning, and led the Bulldogs to a Sugar Bowl win in 2002. Greene spent three years in the NFL after being drafted by the Seattle Seahawks in 2005.

Registration for the tournament will begin at 9 a.m. and will be followed by an autograph session with Ragan and Greene. Food and refreshments will be available throughout the course and a dinner will conclude the day. 

Nephrotic Syndrome and FSGS are conditions that affect the tiny filtering mechanisms in the kidney.  The result is that beneficial protein is spilled from the kidney into the urine and lost.  Over time this condition can lead to kidney failure and the need for dialysis or a kidney transplant.  The cause for Nephrotic Syndrome and FSGS is not known and there is no cure.

The NephCure Foundation is the only organization solely committed to seeking a cause and cure for Nephrotic Syndrome and Focal Segmental Glomerulosclerosis (FSGS).  Comprised of patients, their families and friends, researchers, physicians, and other healthcare professionals, NephCure aims to help science unlock the biological mechanisms that cause these serious conditions and ultimately find a way to cure and prevent them.