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People living with a rare disease are at the center of everything we do at Citrine.We focus on building access to treatments for the 20 million rare disease patients in China who have very few options available to them. We aim to deliver innovative therapies and to make a positive impact on the lives of patients.
We focus on rare neurological, metabolic/endocrine, renal/liver and pediatric oncology diseases.
Rare neurological diseases include developmental defects of the nervous system, genetic and metabolic diseases, neuroimmune diseases, neurological tumors, special types of infectious diseases of the nervous system, neurodegenerative diseases, and neuromuscular diseases. At present, more than 7,000 rare diseases have been found in humans, about 60% of which are neurological diseases.
The endocrine and metabolic system includes endocrine glands and endocrine tissues, which regulate human function by secreting hormones. Endocrine diseases are rare and often very difficult to diagnose and treat. Among the 121 rare diseases included on China’s national list of rare diseases in 2018, more than 20 are endocrine/metabolic-related rare diseases.
Chronic kidney disease (CKD) is a non-communicable disease usually caused by diabetes and hypertension. The prevalence of chronic kidney disease in China is reported to be 10.8%, and is predicted to increase in the next decade, especially among rural regions and certain geographic regions.
Liver disease accounts for approximately 2 million deaths per year worldwide, with 1 million due to complications of cirrhosis and 1 million due to viral hepatitis and hepatocellular carcinoma.
Specific rare liver and kidney diseases often associated with metabolic syndrome as well as viral hepatitis.
In recent years, the incidence of childhood cancer has been on the rise, and malignant tumor has become one of the main causes of death among adolescents. More than 250,000 children worldwide are diagnosed with cancer each year, and about 90,000 children die of cancer—making it the leading cause of death among children. In addition to leukemia, malignant lymphoma and other blood tumors, childhood tumors often impact the central nervous system.