Tuesday, May 17, 2011

An investigation to determine the efficacy and safety of lentivirus mediated FXN gene delivery for the correction of Friedreich ataxia

Principal researchers: Dr Mark Pook and Dr Michael Themis Brunel University,
Uxbridge, UK

In children with Friedreich ataxia, muscle and ataxia parameters are associated

Developmental Medicine & Child Neurology, 53: 529–534. doi: 10.1111/j.1469-8749.2011.03931.x (Article first published online: 16 MAY 2011)

SIVAL, D. A., POUWELS, M. E., VAN BREDERODE, A., MAURITS, N. M., VERSCHUUREN-BEMELMANS, C. C., BRUNT, E. R., DU MARCHIE SARVAAS, G. J., VERBEEK, R. J., BROUWER, O. F. and VAN DER HOEVEN, J. H. (2011).

Keywords: Friedreich ataxia (FRDA), ataxia, International Cooperative Ataxia Rating Scale (ICARS), muscle weakness, muscle ultrasound density (MUD), muscle force, sensory evoked potentials, and ICARS scores, FXN gene analysis, persistently absent sensory evoked potentials, leg muscle force.