Advocacy in Action: More Funding for FSGS Research! June 16, 2015 by Kylie Karley Remember When… Back in January, FSGS was officially added to the list of conditions eligible for research funding through the Department of Defense’s 2015 Peer Reviewed Medical Research Program (PRMRP). This opened up a new $247.5 million funding source for FSGS researchers. When NKI and our patient families visited Washington DC earlier this year, we advocated to keep FSGS on the list and to increase the funding available for research. More Good News: Last week, the Senate Appropriations Committee approved the 2016 Defense Appropriations budget including $31.2 million in additional funding for the Peer Reviewed Medical Research Program. FSGS made the list of eligible conditions again, so if the bill passes without changes, our researchers will have access to $278.7 million in 2016! What’s Next? The bill will have to be passed by the House and Senate and signed by the President before going into effect. We’ll be tracking it and keeping you updated as it moves through the legislative process. Thank You! Increased funding for research and including FSGS on the list of eligible DOD conditions were two of our “Asks” when we visited Capitol Hill. Thank you advocating in person, writing letters and emails, and making phone calls to increase government awareness of Nephrotic Syndrome diseases and the vital role that government funding plays in finding better treatments and cures! Keep it up and stay tuned for more opportunities to be an Advocate!
NEW Funding Source for FSGS January 9, 2015 by Kylie Karley President Obama signed the appropriations package to fund federal agencies through fiscal year 2015 (ending September 30, 2015). This includes allocations for NIH and other health care funding agencies such as the Department of Defense (DoD), which oversees the Peer-Reviewed Medical Research Program (PRMRP). For the first time, FSGS is listed as a condition eligible to study in the PRMRP. Because FSGS is listed, FSGS researchers have an opportunity to compete for grants from a NEW funding source. This is HUGE. This opportunity is a direct result of NephCure’s professional and volunteer advocacy work in Washington, D.C. and the efforts of our staff to work collaboratively with scientific partners at NIH and the VA to highlight the benefits of studying this disease through the DoD program. The total amount in the PRMRP program is $247.5 MILLION! UPDATE: The Program Announcement was released April 17 for the 2015 cycle. There are five funding mechanisms available. Click here for details.
FSGS and Pregnancy Expanding your family is exciting, but how will this affect your health when you are living with FSGS? Carrying a child puts a lot of additional stress on your body, so it is important to discuss pregnancy with your doctor beforehand. FSGS and Pregnancy Webinar Pregnancy and FSGS: Laci Weatherford’s Story Glomerular Disease and Pregnancy ACKD Research Article
NephCure Announces Inaugural APOL1 Kidney Disease Awareness Day on April 30th April 11, 2024 by Imanté Eichelberger Leave a Comment NephCure, a leading nonprofit organization dedicated to finding better treatments and a cure for rare kidney disease, is proud to announce the inaugural National APOL1 Kidney Disease Awareness Day on April 30th, 2024.The day is dedicated to raising awareness of APOL1 kidney disease, a genetic form of kidney disease that predominantly impacts people of African descent. APOL1 kidney disease is caused by specific genetic variations, or changes, in the APOL1 gene, which can lead to a significantly higher risk of developing kidney disease. Individuals who inherit these gene variations have 10 to 30 times the risk of developing kidney disease, making it crucial to raise awareness and support those affected by this condition. NephCure invites everyone to join in on the movement and help raise awareness for APOL1 kidney disease by supporting the following: How you can help: Share on Social Media: Spread the word by sharing your photos and posting the downloadable APOL1 kidney disease awareness graphics on social media. Remember to use the hashtag #HopeForAPOL1 and tag @NephCure. Attend the APOL1 Day Virtual Town Hall: Join NephCure on April 30th at 7pm ET as they host an APOL1 Day Virtual Town Hall. Register for this FREE webinar where you can connect with leading APOL1 researchers, doctors, and individuals personally affected by APOL1 kidney disease. Did you Know? 13% of African Americans carry APOL1 gene changes that can lead to APOL1 kidney disease. Of these individuals, 1 in 5 will develop APOL1 kidney disease. APOL1 kidney disease can impact individuals of all ages, including children and young adults, often progressing swiftly towards kidney failure. Unfortunately, there are currently no FDA-approved treatments specifically for APOL1 kidney disease. Approximately 40% of cases of kidney failure in those without diabetes are associated with APOL1 gene variations. NephCure thanks Vertex Pharmaceuticals for their generous support in sponsoring this event and making APOL1 Kidney Disease Awareness Day possible. For more information about APOL1 Awareness Day, please visit resources.nephcure.org/apol1 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 – PARASOL Grant Program NephCure – PARASOL Grant Program Objectives NephCure in cooperation with the International Society of Glomerular Diseases, the Kidney Health Initiative, and the National Kidney Foundation announces a research grant program to support the PARASOL project. PARASOL is a collaborative international effort that aims to define the quantitative relationships between short-term changes in biomarkers (proteinuria and GFR) and long-term outcomes in order to support the use of alternative proteinuria-based endpoints as a basis for accelerated and traditional approval. The PARASOL group will achieve this in 2024 by conducting a large-scale analysis of existing data from patients of all ages with FSGS who have participated in observational cohort studies, completed clinical trials, regional or national registries, or real-world data sets. Meritorious Characteristics Proposals will be evaluated for their compliance with the guiding principles described above. The following project characteristics will be considered meritorious if: The proposed project is willing and able contribute necessary and valuable data to the PARASOL project to support achievement of PARASOL aims. The proposed project leads are able to meet the timelines established for PARASOL project success. Eligibility The program is open to all investigators or clinicians who oversee an existing cohort study, registry, or real-world data inclusive of substantial FSGS patient data meeting the dataset eligibility criteria* below: Dataset Eligibility Ability to define cohort based on eligibility criteria below Proteinuria and serum creatinine at biopsy/diagnosis and longitudinally. Ascertainment of kidney failure and death per definitions above. At least 100 participants meeting inclusion criteria in Table 2 AND: ≥24 months of follow-up ≥3 UPCR measurements 6-24 months post-index UPCR (any) ≥3 eGFR measurements 6-24 months post-index UPCR in prior bullet Dataset-Required Criterion Definition Biopsy-proven or monogenic FSGS Documented description of adjudication of FSGS diagnosis Index UPCR≥1 g/g (or equivalent g/day) At least one UPCR≥1 g/g at or after biopsy/diagnosis (or up to 3 months prior to biopsy/diagnosis) Index eGFR≥30 ml/min/1.73m2 eGFR measurement must be within ±3 months from index UPCR UPCR measurement 6-24 months after index date ≥1 UPCR measurement 6-24 months after index UPCR No evidence of kidney failure (see above definition) within 6 months No kidney failure events prior to the first observed UPCR>6 months post-index date *Note that data from all participants meeting inclusion criteria in Table 2 will be requested/analyzed, however, at least 100 participants must meet the additional requirements above. Funding NephCure will award funds in accordance with anticipated costs per project to support high quality dataset contributions to PARASOL. To protect the integrity of the PARASOL study and ensure regulatory compliance, applying registries must be reviewed and approved by the PARASOL Organizing Committee before submission of a proposal for funding from NephCure. Projects will be funded directly by NephCure to the recipient institutions. Budget and justification will be reviewed by the PARASOL Finance Committee in consultation with the PARASOL Organizing Committee and decision will be made regarding support needed to ensure high quality registry data is able to be contributed. Funds will be provided to cover project direct costs only. No indirect costs.NephCure has a long standing policy to support direct costs only. The number of projects funded during will be dictated by the size of project budgets requested and awarded. NephCure makes this determination at the time of review. Application/Review Process The process of submitting applications to NephCure for this grant program occurs in 2 steps. No application can be submitted to NephCure before approval of/invitation by the PARASOL Organizing Committee, which assesses proposals for feasibility. PARASOL Organizing Committee Approval The investigator meets with the PARASOL intake team to discuss PARASOL objectives and alignment and feasibility of including the proposed registry/dataset. An assessment of contributing registry/dataset value is provided to the PARASOL Organizing Committee. Approval and invitation to submit is necessary prior to submission for funding. Grant Submission to NephCure Once the PARASOL Organizing Committee approves the proposed registry/dataset, the investigator will be invited to submit a grant proposal to NephCure utilizing the application template provided. NephCure is responsible for funding decisions. Applicants may discuss the application process with Dr. Laura Mariani lmariani@med.umich.edu prior to making an application. Application Format Please complete and submit using the PARASOL grant application template. Terms of Support Funds (direct costs only) are provided to investigator’s institution for use by the applicant; it is the applicant institution’s obligation to ensure proper use of funds and timely submission of progress reports. Funds will be provided to cover project direct costs only. No indirect costs. NephCure has a long standing policy to support direct costs only. Disbursal of grant funds will be made in 2 payments. 50% of payment will be made upon notice of award and remaining 50% will be paid following receipt of registry data in format established by PARASOL Data Analysis Committee. Principal investigators must comply with human institutional review board requirements and demonstrate current approval of these committees. It is expected that data provided to the coordinating center will be shared in a de-identified manner with contributing registries within PARASOL. It is expected that publications will be submitted in compliance with the publications policies of PARASOL. NephCure support will be acknowledged by grantees in publications, presentations, abstracts, and other relevant press releases and on associated websites. NephCure does not discriminate on the basis of race, gender, religious, or ethnic group; applicants should assure compliance with this policy in designing and executing their studies.
Rare Kidneys Connect – Toronto April 3, 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
Countdown to a Cure NYC – 20th Anniversary March 26, 2024 by Hillary Kent Leave a Comment Save the Date: Wednesday, October 30, 2024 for the 20th annual Countdown to a Cure NYC — an incredible night of dinner, drinks, silent and live auctions, connecting with patients and other community members, and networking with hundreds of New York’s leading professionals. All proceeds support NephCure’s mission of fighting for better treatments and a cure for Nephrotic Syndrome, FSGS, and other rare, protein-spilling kidney diseases. Registration coming soon.
LA Walk & Patient Education Day February 27, 2024 by Hillary Kent Leave a Comment Join us on Sunday, April 28th at the Annenberg Community Beach House in Santa Monica as our SoCal patient community joins together for a family-friendly day full of fun, food, music, movement, and more! Gather your family and friends and grab your sneakers and sunscreen because we are heading back to the beach for more fun in the sun! Learn from the experts as we will be joined by the leading Nephrologist in our region, who will discuss the latest news and developments in nephrotic syndrome, FSGS, IgAN, APOL1, and more. And new this year, the leading pharmaceutical companies who are working tirelessly to bring new RKD treatments to market will be onsite to share news and information about clinical trials in progress. Lunch and Learn: We are thrilled to announce Dr. Elaine Kamil will be back to moderate a panel of local leading pediatric and adult nephrologists, who will be announced in the coming weeks! Dr. Kamil is the former Director of Pediatric Nephrology at Cedars-Sinai Medical Center, and Emeritus Professor of Pediatrics at Cedars-Sinai, where she serves as the principal investigator on several glomerular diseases clinical trials. Register today and help us walk towards a cure!
Rare Kidneys Connect – Charlotte February 26, 2024 by Hillary Kent Join NephCure, along with fellow patients and caregivers affected by rare kidney diseases, in Charlotte 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
Rare Kidneys Connect – Dallas January 26, 2024 by Hillary Kent Join NephCure, along with fellow patients and caregivers affected by rare kidney diseases, in Dallas 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