Nephcure Northeast: Information Center

If you are a patient living with rare kidney disease, one way you can help advance kidney care is by taking part in a clinical trial. Having more participants in a clinical trials means a greater chance of new treatments making it to the market!

The EPPIK Clinical Study for Children with FSGS and MCD (Lake Success, NY) - Read more about The EPPIK Clinical Study for Children with FSGS and MCD (Lake Success, NY) on NephCure.

https://nephcure.org/get-involved/events/rare-kidneys-on-the-hill-day/

Rare Kidneys on the Hill Day Rare Kidneys on the Hill Day Washington, DC Rare Kidneys on the Hill Day is an opportunity for advocates to raise awareness about rare kidney disease - Read more about Rare Kidneys on the Hill Day on NephCure.

For parents of children with rare kidney diseases Online resources for parents

There is no cure for rare protein-spilling kidney diseases (yet). But as a patient YOU can help move the research forward - for better treatments and ultimately - a CURE!

Please consider taking part in one of the many clinical trials available. You can find more information at the link below:

Clinical Trials Clinical Trials By participating in a clinical trial, you not only gain access to cutting-edge treatment & expert care, but you are participating in a potential medical breakthrough that could help many more people in the future. Throughout the trial, you’ll be under the careful watch of leading p...

Did you get a chance to check out NephCure's new website?! Get ready to dive into our comprehensive Resource Hub, designed to empower and support you on your rare kidney disease (RKD) journey like never before!

Say goodbye to endless scrolling and hello to efficiency! Our intuitive search filters allow you to explore our vast collection of resources with ease. Filter by resource type, disease type, topic, and more to find exactly what you need, when you need it.

From informative articles and expert guides, to engaging videos and practical toolkits, our Resource Hub is your go-to destination for all things RKD-related. Knowledge is power, and we're here to arm you with the information you deserve.

Visit NephCure.org today and start exploring our Resource Hub!

https://nephcure.org/support/resources/

Congratulations and best of luck going forward Rick!

Recipient of world's first pig kidney transplant discharged from Boston hospital The recipient of the world's first pig kidney transplant​ is heading home from Massachusetts General Hospital Wednesday, nearly two weeks after the surgery.

A major step was taken over the weekend right here in Boston. The first xeno-transplant of a pig kidney into a live human recipient.

Surgeons in Boston transplant pig kidney into a patient, a medical milestone The man continues to improve, doctors said. Organs from genetically engineered pigs one day may make dialysis obsolete.

https://nephcure.org/events/parent-support-group/

NephCure Parent Support Group - NephCure Join us for an informal time to connect with other parents of children with rare, protein-spilling kidney disease.

Join us for the groundbreaking webinar, 'Are You Prepared for Prepare-NS?'

Discover the pioneering efforts of the FDA-funded Prepare-NS study on January 25th at 1pm EST. Dive into the development of patient-reported and caregiver-reported questionnaires aimed at measuring the impacts of edema (aka swelling).

Explore key discussions:

🔹The impact of edema on progressive disease
🔹Health questionnaire development by Prepare-NS
🔹Patient and caregiver-reported measures
🔹Personal experiences shared by patients
🔹Insights from pediatric and adult nephrology experts
🔹Commentary on patient-focused drug development

By the end of the webinar, you'll be well-versed in patient-focused approaches to capturing edema in nephrotic syndrome for clinical trials!

Secure your spot now! Click the link below to register for the FREE webinar.

https://www.is-gd.org/webinar-are-you-prepared-for-prepare-ns

Behind all the work, events, fundraisers, and support, NephCure’s staff team is constantly working hard to ensure we do the best for our patient community, especially assisting patients in navigating their disease journey, including assisting with healthcare access, school support services (such as IEPs and 504 plans), insurance, medication assistance programs, and more, which is why we are highlighting Montrez Lucas!

Montrez Lucas, LMSW joined NephCure in October 2022 as the Associate Director of Patient Navigation. He is a licensed social worker with an extensive background in the field of healthcare social services. Before coming to NephCure, he led a team of social workers at Emory Healthcare in Atlanta.

In his role at NephCure, Montrez helps patients, caregivers, and care partners navigate through healthcare services and systems. He builds relationships with rare kidney disease patients and families to help identify and solve problems, locate resources, reduce health disparities, empower patients to get the care they need, and support positive health behaviors and outcomes.

Many in our community has been assisted by Montrez. We encourage you to share your favorite memory or a story of how he has impacted you or your family!

If you haven’t signed up for our monthly newsletter, you’re missing out!

Stay in the loop with NephCure’s monthly newsletter, and sign up for a front-row seat to important educational resources, exciting news, upcoming events, and more!

Be the first to access important resources, and stay informed on the latest happenings and research in the rare kidney disease space! Don’t miss out on a chance to be a part of our community and dive into information surrounding rare kidney diseases!

Click the link below to sign up today!

https://nephcure.org/join-nephcure-4/

Reminder:
This Tuesday January 23rd beginning at 8:00 PM, the Grandparent Support Group will be meeting up online.
Please click the link below for more information and to register.

NephCure Grandparent Support Group - NephCure Join us for an informal time for grandparents of those who are living with rare kidney diseases to connect, held on the fourth Tuesday of each month at 8 p.m. EST.

Reminder:
The Nephcure Teen Support Group will be taking place this Monday January 22nd at 7:30.
Please click on the link below for more information and to register!

NephCure Teens Support Group - NephCure NephCure Teens is a monthly virtual support gathering just for teens, ages 13-17, with rare, protein-spilling kidney disease. Join us every fourth Monday of the month at 7:30 p.m. ET. These support groups are moderated by NephCure patient support volunteers and/or NephCure staff.

https://nephcure.org/2023/12/nephcure-summit-2024-empowering-lives-connecting-hearts/

Secure Your Spot Today: NephCure Patient Summit 2024 - NephCure Join us in making the season brighter for those in need. Your generosity can make a life-changing impact. Support our mission to find a cure.

Sean Converse, an IgAN patient, crossed paths with Dr. Leonardo Riella at the 2023 Boston Countdown to a Cure gala, where they were honored as the Fund a Cure family and Medical Professional award recipient, respectively.

Their connection sparked a special relationship, and last month, Sean and his mom got an exclusive behind the scenes look at Dr. Riella's lab! 🔬

Sean and his mom even got to join a staff meeting at Dr. Riella’s lab to better understand the immune response at a cellular level.

But it didn't stop there – they got an inside scoop on the process of research grants and funding, understanding how critical decisions are made for allocating funds.

And guess what? Dr. Riella is going above and beyond by conducting genetic testing for Sean! 🧬

The pursuit of answers continues, and NephCure events are the catalyst for these life-changing connections. Don’t miss out on events happening in your area, you never know who you will meet that will help you in your rare kidney disease journey.

To see a full list of events, visit https://nephcure.org/events!

New Era of Preventing End-Stage Kidney Disease Act H.R.6790 - NephCure Revolutionize rare kidney disease care, reduce costs, empower patients. This act is for early detection, innovative treatments, and better lives.

https://www.calliditas.se/en/calliditas-therapeutics-announces-full-fda-approval-of-tarpeyo-the-only-fda-approved-treatment-for-iga-nephropathy-to-significantly-reduce-the-loss-of-kidney-function/?fbclid=IwAR0SpH_VNPFUuJMfBG57QX8evnuE8wwutQaJMWB1r9eWLbmDopvblfPCPRw

Calliditas Therapeutics announces full FDA approval of TARPEYO®, the only FDA-approved treatment for IgA nephropathy to significantly reduce the loss of kidney function - Calliditas Therapeutics AB Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) (“Calliditas”), today announced that the U.S. Food and Drug Administration (FDA) has approved TARPEYO (budesonide) delayed release capsules to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy...

https://nephcure.org/events/adult-support-group/

NephCure Adult Support Group - NephCure Join us for an informal time to connect with other adult patients living with rare, protein-spilling kidney diseases on the second Thursday of each month at 8 p.m. ET. These support groups are moderated by NephCure patient support volunteers and/or NephCure staff.

Join us on Thursday, October 19th, at 3 pm ET during a special live Q&A with NephCure to showcase Dongwon Lee PhD, Assistant Professor of Pediatrics Harvard Medical School and Principal Investigator of the Dongwon Lee Lab Boston Children’s Hospital and his work with the Sampson Lab for Kidney Genomics.

Dongwon is a computational biologist and human geneticist with expertise in machine learning. The Lee and Sampson Lab’s research uses genomics to better understand, treat, and ultimately cure nephrotic syndrome. Through genomics, researchers can decode the genetic instructions within our cells, pinpointing the specific changes that lead to disease. Eventually, it may allow us to develop targeted treatments tailored to the genetic causes of each patient’s condition.

In partnership between NephCure and Sampson Lab for Kidney Genomics, we are building a collaborative effort to highlight important research and resources to assist rare kidney disease patients on their journey.

We will host our Q&A on Zoom (meeting link in bio) to discuss the genomics of kidney disease, current research, and how it contributes to the search for a cure.

When she was four years old, Celest Foo was diagnosed with a rare disease known as focal segmental glomerulosclerosis (FSGS) - a condition in which the filters in her kidneys become scarred and are unable to filter blood.

This diagnosis led to a 15-year fight to maintain functional kidneys with medication under the care of our teams from National University Hospital - NUH and National University Centre for Organ Transplantation (NUCOT).

However, Celest’s kidneys eventually failed in January 2020, when she was 19 years old. As a result, she had to undergo haemodialysis two to three times a week, for five hours each time.

Celest’s father, Mr Sam Foo, could not bear to see his daughter in such a constantly weak state. He knew that a kidney transplant would be the only way to restore Celest’s quality of life. So, when tests revealed that he was a suitable donor for his daughter, Mr Foo did not hesitate to donate his kidney.

Read the full story: https://for.sg/nuhs-dads-what-i-did-for-love

Read how another father overcame his aversion to seeing the doctors, so that he could receive treatments for his depression and be eligible to donate his kidney to his daughter: https://for.sg/nuhs-76-year-old-father-saves-beloved-daughter

Happy Father’s Day to all the amazing dads in our lives ❤️

https://www.reuters.com/markets/deals/novartis-acquire-us-based-chinook-32-bln-upfront-2023-06-12/?fbclid=IwAR02JgYyWZdtQpP5HW05z01sAbvfNY0rw4PkrjFyZyoL00Pov2EW95dZc4k

Novartis buys Chinook for $3.5 billion in race to treat rare kidney disease Novartis said it has agreed to acquire U.S. biotech firm Chinook Therapeutics for up to $3.5 billion to boost its late-stage drug development line-up, raising the stakes in the race for a rare kidney disease treatment.

Rare Kidneys On the Hill Day is back in-person this summer, and we need your support for our community to get access to better care and treatments!

On July 11-12, 2023, in Washington, D.C., join NephCure on Capitol Hill and meet with your congressperson to help advance legislative and policy goals that will support the rare kidney disease (RKD) community.

Be a part of this fundamental advocacy effort to directly educate decision makers about RKD and ask their support for critical fiscal year (FY) 2024 policies to increase funding for RKD research and improved health services and treatments.

Our goals include increasing funding for vital needs such as research and patient access to necessary treatments, improved diagnostics, expert care, community health services, and more.

Right now, congress and other government officials are considering health policy appropriations funding and pieces of legislation that directly impact critical programs to advance RKD treatment and care, which is why we need your support.

To learn more and register, by clicking the link below!

https://resources.nephcure.org/rkdw-2023

https://www.youtube.com/watch?v=yoJGuLsNSIw

A New Future for Rare Kidney Disease: One Teen's Journey with FSGS Meet Joshua Albright. A happy and seemingly-healthy 17-year-old who had just graduated from high school, and on his way to college—his whole life ahead of hi...

We're here for you and because of you! When NephCure was first founded in 2000, people living with FSGS, IgAN, and other rare kidney diseases had little reason to hope. There was hardly any research on these diseases, let alone any specific treatments or a cure.

But today, we are set to experience an incredibly rare moment for a rare disease community: we are likely only months away from the announcement of one of the first-ever FDA-approved treatments for rare kidney disease!

This is the moment we’ve all been working toward. Your support has made this success possible.

So thank you, thank you, THANK YOU to our incredible community of supporters who have volunteered, donated, spread the word, held fundraisers, and, most importantly, participated in the clinical research. Your contributions are truly making a difference!

From our family to yours, have a happy, hearty, and healthy Thanksgiving.

NephCure Celebrates Largest Advocacy Event To Date - NephCure Kidney International ® NephCure and the rare kidney disease community celebrate the success of Tuesday’s Rare Kidneys on the Hill Day. Rare Kidneys on the Hill Day, which takes place during NephCure’s Rare Kidney Disease Week, serves as the premier advocacy event for the rare kidney disease community.

Vertex Announces Inaxaplin (VX-147) Granted Breakthrough Therapy Designation by U.S. FDA and Priority Medicines (PRIME) Designation by the EMA Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has granted inaxaplin (VX-147) Breakthr

https://www.sciencedaily.com/releases/2022/05/220505085618.htm

Combining certain meds with ibuprofen can permanently injure kidneys: Commonly prescribed hypertension drugs may be harmful in combination with ibuprofen Anyone who is taking a diuretic and a renin-angiotensin system (RSA) inhibitor for high blood pressure should be cautious about also taking ibuprofen, according to new research.

https://www.youtube.com/watch?v=CI4Y_h3tfvs

NephCure U: Cutting through the Noise: An Inside Look at IgA Nephropathy Get the Facts on IgA Nephropathy, or IgA Kidney Disease, from Dr. Sean Barbour. Dr. Barbour will give us an inside look on the latest research and how you ca...