Kidneys are the vital organ of the body that flushes out waste from the body and cleans the blood. However, some renal diseases can impact the function of the kidney and other organs, too. Kidney diseases can lower the ability to clean your blood, which results in the accumulation of waste in the blood—it may result in serious consequences such as kidney failure, organ failure, and metabolic disorders. It is worth highlighting here that 60-80 individuals per 100,000 cases in the United States and Europe have been reported of rare kidney diseases. These rare renal disorders are often related to genetic abnormalities and are the leading cause of diabetes. If an individual remains undiagnosed for a longer time and does not receive treatment over time, it can be fatal, too.

    The available treatment options, such as dialysis, biopsy, and kidney transplants, are complex and may require frequent visits in healthcare settings. Driven by the complexities associated with treatment options, the rare kidney disease market players are emphasizing the development of advanced therapeutic drugs to treat a broad spectrum of diseases. It is worth noting here that pharmaceutical companies have developed several drugs that have received approval from the FDA to treat kidney diseases. These drugs includes Atrasentan, Bardoxolone Methyl, Benlysta, Cosentyx®, Cozaar®, Crovalimab, Cystadrops®, Empaveli®, Galafold®, Ianalumab, Iptacopan, Lupkynis™, Narsoplimab, Pegunigalsidase Alfa, Rituxan®, Saphnelo™, Sibnayal™, Sparsentan, Tarpeyo®, Thiola® and Ultomiris®. However, for an effective treatment, it is essential to make people aware of rare kidney disorders, their consequences, and available treatment options. In this context, let’s read about different types of rare kidney diseases that can lead to harmful effects on humans.

    Types of Rare Kidney Diseases

    It is interesting to highlight here that, since 2020, more than USD 4.5 billion of capital investment has been made by rare kidney disease market leaders to develop novel drugs for the treatment of patients suffering from the below-listed diseases.

    1.     AIport Syndrome

    It is a form of genetic disorder that occurs due to the malfunctioning of genes that control the shape of the body parts. When these genes are not functional, parts such as eyes, ears, and kidneys do not develop accurately. The syndrome may result in loss of hearing and eye problems and cause severe impact on the kidneys.

    1.     Amyloidosis

    This condition can cause the amyloid protein to build up inside the body’s organs and tissues. The clump formation of these proteins in the body can cause damage to the kidney, intestine, liver, brain, and heart. Consequently, there are two forms of amyloidosis that can affect kidneys, namely primary amyloidosis and dialysis-related amyloidosis. Primary amyloidosis results in kidney diseases, and dialysis-related amyloidosis occurs when an individual is on dialysis for a long time. Both these types of amyloidosis are curable with treatment options available and also through drug development by rare kidney disease market players.

    1.     APOL 1-Mediated Kidney Disease

    It is a range of rare kidney diseases associated with mutations within the apolipoprotein L1 gene. There are two copies of these genes present in humans, however individuals with mutated genes are at higher risk of having genetic related kidney disease. This APOL 1-Mediated kidney disease may lead to the failure of kidneys and organs at a relatively faster rate. The discovery of the gene variant has been made in recent years, so awareness of this rare kidney disease is low among people. The rare kidney disease market players are emphasizing raising awareness about this type of disease to help people understand the consequences associated with the condition. In this context, the American Kidney Fund (a profound organization) is launching the first annual APOL 1-Mediated Kidney Disease Awareness Day on April 30, 2024, to spread knowledge about the disease and available treatment options.

    1.     Cystinosis

    It is a rare form of multisystem genetic disease that can cause kidney failure in children, teens, and adults. Cystinosis generally occurs due to mutations within CTNS genes. The condition develops when cysteine (a protein component) builds up inside the cells and tissues. The excessive accumulation of cysteine can cause damage to the kidney, pancreas, and brain function. Currently, rare kidney disease market players are focusing on the development of target-specific therapies that can help treat these conditions so patients can live a quality life.

    1.     Hemolytic Uremic Syndrome

    This condition usually occurs when red blood cells are completely damaged and block the kidney filters. It generally occurs due to E. coli bacteria infection and viruses, as well as inherited gene mutation. The rare disease generally impacts children, and the symptoms of this disease are similar to those of stomach virus infection as the infection persists into the body. Hemolytic uremic syndrome will be serious, and it may result in acute kidney injury that further leads to organ failure and rare diseases.

    1.     IgA Nephropathy (IgAN)

    It is a form of autoimmune disorder, also referred to as Berger’s disease, that occurs due to improper functioning of the immune system. IgAN over-secretion can result in abnormal antibodies in the kidney that can trigger inflammation and reduce the kidney’s ability to filter blood from the bloodstream. The condition causes severe damage to the kidney and often leads to kidney failure.

    1.     Lupus Nephritis

    Lupus is an autoimmune disease that occurs when the immune system does not operate effectively, reducing the body’s capacity to fight infection. It might also trigger the immune system to target healthy cells. Lupus nephritis is a condition in which the immune system attacks the kidneys. The most severe form of lupus nephritis is proliferative nephritis, which can permanently destroy the kidneys. Many scientists believe that lupus is caused by a mix of hereditary and environmental causes.

    Concluding Remarks

    In conclusion, rare kidney diseases can be fatal if not diagnosed early or if the patient does not receive treatment on time. Hence, the patient needs to understand the types of rare kidney diseases and their potential impacts. With growing awareness about the negative impact of these conditions, several rare kidney diseases market players are increasing their efforts to develop innovative drugs that can address these disorders and save people from consequences. Currently, several drugs are under clinical trial pipeline that can address these rare kidney disorders. The growing pipeline of therapeutic drugs and rising funding activities in the field indicate the bright future of the market and may result in potential treatment options that can save patients.

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