A Novel Therapeutic Approach for Treating Rett Syndrome.

Abstract

Background: Rett syndrome is a rare X-linked dominant neuro-developmental and pervasive developmental disorder that has no satisfactory or effective treatment. The diagnosis is generally clinical and autistic feature is a typical finding in all patients. We have previously described the short-term treatment of an Iraqi girl with Rett syndrome who was first seen at the age of about three years. The girl was treated with intramuscular cerebrolysin and oral citicoline for 40 days. Treatment was associated with significant improvement with the development of purposeful movement and the ability to hold feeding bottle with assistant of the mother and feed her. She was able to stand and step one step holding furniture. She started babbling and showed some reduction in the autistic features. The aim of this paper is to describe the extended treatment of the girl with Rett syndrome which was treated with a novel therapeutic approach which included intramuscular cerebrolysin, citicoline and piracetam. Patients and methods: After the initial 40 days treatment, the girl received several treatment courses that included intramuscular cerebrolysin, citicoline and piracetam. Results: Several months of treatment resulted in improvements in behavior, autistic features and mobility and she was walking confidently holding a wall or furniture. Treatment was not associated with any side effects. Conclusion: Rett syndrome is a very complex neuro-developmental and pervasive developmental disorder that has no satisfactory or effective treatment. The use of novel therapeutic approach which included intramuscular cerebrolysin, citicoline and piracetam for the treatment of Rett syndrome resulted in within few months a significant improvement that have not been reported without treatment or with any other therapies before.