According to a story from Bioportfolio, the biopharmaceutical company Newron Pharmaceuticals has chosen to support Rett Syndrome Awareness Month this year. Rett Syndrome Awareness Months falls on the October of each year. Before the month began, the company participated in an international conference called “Rett Syndrome Research, Towards the Future.” Newron is dedicated to the development of all new, innovative therapies for diseases that affect the central nervous system.
About Rett Syndrome
Rett syndrome is a brain disorder that become evident early in the lives of its female patients. The disease is caused by a genetic mutation that affects the MECP2 gene. This gene is found on the X chromosome. Boys who have this mutation die soon after being born, so Rett syndrome exclusively affects girls. It occurs as a spontaneous mutation in the vast majority of cases, and is rarely inherited from parents. Symptoms include sleeping issues, difficulty speaking, poor coordination, scoliosis, seizures, small head size, slow growth, and repetitive movements. There is no cure for Rett syndrome, and management focuses on maintaining function and alleviating symptoms. Life expectancy for patients is around 40 years. Death often occurs sponatenously, and is often linked to brainstem malfunction, gastric perforation, or cardiac arrest. To learn more about Rett syndrome, click here.
Rett Syndrome Research From Newron
The conference touched on the latest in Rett syndrome research and also focused on the perspectives of patient families and caregivers. Newron also presented the latest update on an international Burden of Disease (BOD) study. A burden of disease study is intended to measure the costs of Rett syndrome, including information about the financial and human cost of the disease. Such a study incorporates the perspectives of health workers, physicians, and care givers.
In addition, Newron is also in the middle of a Phase 3 clinical trial testing Saritozan as a treatment for apnea (abnormal breathing) associated with Rett syndrome. Instances of labored or irregular breathing in patients with the syndrome can be a potentially dangerous complication that requires frequent monitoring on the part of caregivers. More details about the data from this research will be presented to the public at the end of the month.