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If so, they may be eligible to participate in a research study on a new investigational medication for children and adolescent females aged 5-15 with Rett syndrome.<\/p>\n
Rett syndrome is a genetically determined neurological disorder, which is characterized by arrested development, regression of acquired skills, loss of purposeful use of the hands and speech, stereotypical movements, and intellectual disability.<\/p>\n
Currently, treatment for Rett syndrome focuses on the management of specific symptoms and supportive care.<\/p>\n
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This study investigates a novel oral medication called \u201ctrofinetide\u201d (also known as NNZ-2566), which has a unique way of acting relative to other drugs that are being tested for Rett syndrome.<\/p>\n
The purpose of the study is to look at the safety and tolerability of trofinetide in children and adolescents with Rett syndrome. The study will also investigate the effectiveness of trofinetide during treatment.<\/p>\n
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This study investigates trofinetide, a modified version of a biologically active part of Insulin-like Growth Factor (IGF-1). IGF-1 is a protein in the human body that helps cells grow. Trofinetide is a modified version of a small part of IGF-1 called GPE, which detaches from IGF-1 in the brain. IGF-1 and GPE play a significant role in regulating the development of the brain and maintaining healthy brain function.<\/p>\n
Trofinetide may help to improve brain function by reducing inflammation, regulating the functioning of cells called microglia, normalising the connections between brain cells and restoring depressed levels of IGF-1 in the brain.<\/p>\n
Click on the image or the link below to access a video about the science of trofinetide;<\/p>\n