Rett Syndrome – Chapter 14: Treatments for Rett Syndrome (ebook)


ChapterĀ 14 – Treatments for Rett Syndrome: Prospects for Targeted Therapies

Although we have gained many insights into the disorder and the functions of associated genes in the half century since the first clinical diagnosis of RTT, we have not, as yet, been able to find therapeutic agents that have the capacity to cure or even substantially ameliorate the disease in humans. Recent trials using mouse models, however, show that it is possible to reverse aspects of the RTT phenotype, as well as therapies aimed at downstream targets of MeCP2, which has greatly increased optimism that human therapeutic trials now being implemented raise the prospect that RTT could be a curable childhood neurological disorder.

About the book

Among the vast body of literature that has grown around Rett syndrome, this volume is the first to be aimed at both clinicians and researchers. It presents a comprehensive overview of the disorder and examines the areas where gaps in knowledge are most significant. Rett Syndrome is intended to be a guide for both initial examination and in-depth study of the disorder. It is a practical text for the physician approaching the disorder for the first time and a valuable reference resource for the specialist or researcher.