Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Neurology

Rett syndrome

Methyl CpG binding protein 2 (MECP2; RTT)

Mouse studies suggest ketamine could help treat Rett syndrome. In a Mecp2 knockout mouse model of Rett syndrome, ketamine rescued sensorimotor dysfunction and markers of disease, whereas vehicle did not. Next steps were undisclosed but could include evaluating ketamine in patients with Rett syndrome.
Ketamine is a generic drug approved to treat severe and chronic pain.

SciBX 5(39); doi:10.1038/scibx.2012.1036
Published online Oct. 4, 2012

Patent and licensing status undisclosed

Kron, M. et al. J. Neurosci.; published online Oct. 3, 2012;
doi:10.1523/JNEUROSCI.2159-12.2012
Contact: David Katz, Case Western Reserve University, Cleveland, Ohio
e-mail:

david.katz@case.edu