This article mainly reported a potential life-threatening reaction to Glatiramer Acetate in Rett Syndrome. Rett syndrome is an X-linked dominant neurodevelopmental disorder manifesting with severe intellectual disability in females caused by various mutations in the MECP2 gene. Glatiramer acetate is a mixture of synthetic polypeptides composed of four aminoacids, also a well-known and safe multiple sclerosis immune modulator. This study observed fourteen patients with mutation-proven Rett syndrome were recruited for a clinical trial with glatiramer acetate, which had to be stopped because of a severe adverse event. The described severe adverse event can be related to these patients' primary autonomic nervous system dysfunction and three of the patients was in life threatening. So, Glatiramer Acetate’s use in Rett syndrome should be cautiously reconsidered.
Read More
