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10 June 2014 - Just published: autonomic dysfunction in Rett syndrome: role of 5-HT1A agonists.

Rett syndrome is an orphan disorder that has devastating effects on the central nervous system. One of the most severe and life-threatening presentations of this syndrome is brainstem dysfunction, which results in disturbances such as breathing deficits, typified by episodes of breathing cessation intercalated with episodes of hyperventilation or irregular breathing. Serotonin has an important role in promoting regular breathing, regulating mood and alleviating Parkinsonian-like symptoms. Targeting serotonergic function is therefore a promising strategy to manage the symptoms of Rett syndrome.

The present review describes the role of serotonin in Rett syndrome (RTT) based on recent studies in transgenic mice lacking functional MeCP2 protein. Serotonin 5-HT1A agonists (notably F15599, also known as NLX-101) have shown remarkable capacity to improve respiratory function in these mice (see Levitt el al. 2013). In addition, such drugs can have anxiolytic and antidystonic properties which would be anticipated to benefit RTT patients. It is suggested that drugs possessing 5-HT1A agonist properties represent a promising strategy for symptomatic management of Rett syndrome.

 

Adrian Newman-Tancredi has extensive experience of drug discovery of serotonergic agents. For more information, Contact.

 

Pinpointing brainstem mechanisms responsible for autonomic dysfunction in Rett syndrome: therapeutic perspectives for 5-HT1A agonists.
Abdala AP, Bissonnette JM, Newman-Tancredi A.
Front Physiol. 2014 May 30;5:205. doi: 10.3389/fphys.2014.00205. eCollection 2014.