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Ana P. Abdala, John Bissonnette, Adrian Newman-Tancredi

Frontiers in Physiology 5: 205, 2014. doi: 10.3389/fphys.2014.00205

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Rett syndrome is a neurological disorder caused by loss of function of methyl-CpG-binding protein 2 (MeCP2). Reduced function of this ubiquitous transcriptional regulator has a devastating effect on the central nervous system. One of the most severe and life-threatening presentations of this syndrome is brainstem dysfunction, which results in autonomic disturbances such as breathing deficits, typified by episodes of breathing cessation intercalated with episodes of hyperventilation or irregular breathing. Defects in numerous neurotransmitter systems have been observed in Rett syndrome both in animal models and patients. Here we dedicate special attention to serotonin due to its role in promoting regular breathing, increasing vagal tone, regulating mood, alleviating Parkinsonian-like symptoms and potential for therapeutic translation. A promising new symptomatic strategy currently focuses on regulation of serotonergic function using highly selective serotonin type 1A (5-HT1A) "biased agonists". We address this newly emerging therapy for respiratory brainstem dysfunction and challenges for translation with a holistic perspective of Rett syndrome, considering potential mood and motor effects.

Promising pipeline

Neurolixis Inc. has in-licensed early-stage clinical assets (Phase 1 and Phase 2) for repurposing in indications with unmet needs in psychiatric and neurological disorders. Read more...

Research

Neurolixis has been awarded several research grants by private foundations, including Parkinson's UK, the Michael J. Fox Foundation for Parkinson's Research, the Rett Syndrome Research Trust and the International Rett Syndrome FoundationRead more...

Therapeutic focus

Neurolixis is developing clinical phase drugs targeting dyskinesia in Parkinson's disease and breathing deficits in Rett syndrome, a devastating orphan disorder. Read more...