It is estimated that there are more than 7,000 rare diseases, accounting for about 10% of human disorders. Rare diseases are usually chronic, progressive, complicated, involving multiple systems and organs. About 80 percent of rare diseases are genetic in origin and about 50 percent are develop at birth or in childhood. With the increasing attention to rare diseases and the rapid development of precision medicine, great progress has been made in the field of gene identification and molecular diagnosis of rare diseases. However, there is still a lack of effective treatment or therapeutics for patients with rare especially in children. Only 5% of rare diseases are curable and about 30% of children with rare diseases die before the age of five.
This Research Topic aims to bring together recent clinical advances on rare diseases in children, particularly the development of novel therapeutics to improve the care of children with rare diseases. We welcome researchers from all over the world to contribute high-quality submissions in the following areas (but are not limited to):
• Discovery and development of therapeutics for rare diseases in children
• Gene therapy for rare diseases in children and clinical applications
• Development of novel surgical treatment strategies for rare diseases in children
• Novel diagnostic approaches for identification and diagnosis of rare diseases in children
It is estimated that there are more than 7,000 rare diseases, accounting for about 10% of human disorders. Rare diseases are usually chronic, progressive, complicated, involving multiple systems and organs. About 80 percent of rare diseases are genetic in origin and about 50 percent are develop at birth or in childhood. With the increasing attention to rare diseases and the rapid development of precision medicine, great progress has been made in the field of gene identification and molecular diagnosis of rare diseases. However, there is still a lack of effective treatment or therapeutics for patients with rare especially in children. Only 5% of rare diseases are curable and about 30% of children with rare diseases die before the age of five.
This Research Topic aims to bring together recent clinical advances on rare diseases in children, particularly the development of novel therapeutics to improve the care of children with rare diseases. We welcome researchers from all over the world to contribute high-quality submissions in the following areas (but are not limited to):
• Discovery and development of therapeutics for rare diseases in children
• Gene therapy for rare diseases in children and clinical applications
• Development of novel surgical treatment strategies for rare diseases in children
• Novel diagnostic approaches for identification and diagnosis of rare diseases in children