New drug candidates could aid treatment of Rett syndrome

Written by Alice Weatherston

Recent research carried out at Cold Spring Harbor Laboratory (NY, USA) has indicated that the lifespan of male mice models of Rett syndrome can be significantly extended through the use of small-molecule drug candidates. The research, which was published in the Journal of Clinical Investigation, could inform a new approach for the treatment of the disease. “At this time, there is no disease-modifying therapy for Rett syndrome,” lead author Nicholas Tonks (Cold Spring Harbor Laboratory) explained, “and the focus of most current therapies is management of symptoms. Our approach represents a new strategy.” Work carried out across 25 years by...

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