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Treatment Options

Your doctor or primary care provider will decide which medications are right for you based on their evaluation of your condition. The information below is provided for reference only. Before starting or stopping any medication, be sure to discuss it, along with all other drug-related information, with your doctor.

The short-term goal of treatment is to stop protein spillage completely, known as remission, or lower the amount of protein spilling as much as possible. The less protein lost, the better the outcome for the patient. Long-term goals include preventing recurring protein in the urine and protecting kidney function from deterioration.

Click the arrow on the treatment options below to learn more.

ACE Inhibitors

An ACE inhibitor (or angiotensin converting enzyme inhibitor) is a medication used primarily for the treatment of heart, blood vessel, and kidney problems. ACE inhibitors prevent an enzyme in your body from producing angiotensin II.

Acthar

H.P. Acthar Gel, commonly known as Acthar, is a prescription medication that is FDA-approved for the treatment of proteinuria associated with Nephrotic Syndrome.

Angiotensin Receptor Blockers (ARBs)

Angiotensin II receptor blockers (ARBs) help relax blood vessels. This effect lowers the blood pressure and makes it easier for the heart to pump blood.

Cyclophosphamide

Cyclophosphamide (brand name: Cytoxan) is a medication that lowers the body’s natural immunity. The immune system is thought to be involved in causing Nephrotic Syndrome.

Cyclosporine

Cyclosporine is a medicine that lowers the body’s natural immunity. The immune system response is thought to be involved in Nephrotic Syndrome. It is also used to prevent rejection of transplanted organs, such as the kidney, heart, and liver.

FABHALTA® (Iptacopan) 

FABHALTA® targets the alternative complement pathway in order to lower proteinuria. FABHALTA® binds to Factor B of the alternative complement pathway and helps block the effects of IgA Nephropathy on the kidneys.

FILSPARI (sparsentan)

FILSPARI (sparsentan) is a recently FDA approved treatment for IgA nephropathy (IgAN).  There are also studies underway for the use of FILSPARI in other types of rare kidney disease (RKD), such as focal segmental glomerulosclerosis (FSGS). 

Liposorber

The Liposorber® LA-15 System is a blood processing system used outside of the body. This device removes certain lipoproteins from the blood. It’s used in pediatric and adult patients diagnosed with focal segmental glomerulosclerosis (FSGS) either before transplant or post-transplant with FSGS recurrence.

Mycophenolate Mofetil

Mycophenolate (brand names: Cellcept, Myfortic) is a medicine that lowers the body’s natural immunity. The immune system response is thought to be involved in Nephrotic Syndrome. This medicine is prescribed if there are too many relapses of Nephrotic Syndrome.

Obinutuzumab

Obinutuzumab can help you lower the amount of protein you are spilling into your urine, go into complete remission and allow you to stop taking other medications. Obinutuzumab offers an alternative to current immunosuppressive therapies and other monoclonal antibodies for difficult-to-treat rare, protein spilling kidney diseases.

Prednisone

Prednisone is used in the management of multiple conditions or diseases in which the immune system plays an important role. Prednisone works by decreasing the body’s immune system response to various diseases or conditions that produce symptoms.

Rituximab

Rituximab is a medicine that is usually used to treat certain types of cancer. It belongs to a class of medications called monoclonal antibodies. Rituximab also offers an alternative to current immunosuppressive therapies for difficult-to-treat nephrotic syndrome.

SGLT2 Inhibitors

SGLT2 Inhibitors are a class of drug that block the kidneys from reabsorbing glucose. SGLT2 inhibitors are an oral medication (taken by mouth) and can help to slow the progression of kidney disease. They also help to lowering the risk of end stage kidney disease (ESKD).

Tacrolimus

Tacrolimus (brand name: Prograf) is a medicine that lowers the body’s natural immunity. The immune system response is thought to be involved in Nephrotic Syndrome. It is widely used to prevent rejection of transplanted organs, such as the kidney, heart, and liver.

TARPEYO (Budesonide)

TARPEYO (budesonide) is a recently FDA approved treatment for IgA nephropathy. TARPEYO (Budesonide) Delayed Release Capsules works to target the cells in the gut that make IgA.  By targeting these cells, the amount of IgA in the body (and kidneys) is reduced.

Find a Specialist

It is important to work with a nephrologist who specializes in rare kidney disease (RKD) and can provide personalized advice based on your individual health needs and changes in available treatments. NephCure has a curated list of NephCure Specialists nationwide ready to help you.

Support Groups

Created for every phase of your RKD journey, discover a safe and supportive environment to connect with others facing similar challenges. Whether you’re a parent, teen, adult, or grandparent affected by rare kidney diseases, our monthly virtual support groups offer understanding, encouragement, and an opportunity to share experiences.

Latest Resources

Informational Documents

Low Phosphorus Diet Guidelines: For Patients with Rare Kidney Disease

Fact Sheet

Congenital Nephrotic Syndrome & Infantile Nephrotic Syndrome

Video

The History & Innovation of C3G: Patient Summit Seminar with Dr. Corey Cavanaugh

Educational Materials

School Accomodations

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