The purpose of this research program is to develop safe and effective treatments for hereditary neurological disorders. Specific research accomplishments in the past year include the following: (1) completion of a phase 2 study of high dose idebenone treatment for Friedreich's ataxia, and (3) implementation of a phase 2 clinical trial to test the feasibility of dutasteride treatment for spinal and bulbar muscular atrophy (Kennedy's disease). The idebenone trial showed evidence of neurological efficacy with intermediate and high dose treatment in ambulatory subjects. Analysis of other outcome measures is in progress.
| La Pean, Alison; Jeffries, Neal; Grow, Chelsea et al. (2008) Predictors of progression in patients with Friedreich ataxia. Mov Disord 23:2026-32 |
| Di Prospero, Nicholas A; Sumner, Charlotte J; Penzak, Scott R et al. (2007) Safety, tolerability, and pharmacokinetics of high-dose idebenone in patients with Friedreich ataxia. Arch Neurol 64:803-8 |
| Di Prospero, Nicholas A; Baker, Angela; Jeffries, Neal et al. (2007) Neurological effects of high-dose idebenone in patients with Friedreich's ataxia: a randomised, placebo-controlled trial. Lancet Neurol 6:878-86 |
| Sumner, C J; Kolb, S J; Harmison, G G et al. (2006) SMN mRNA and protein levels in peripheral blood: biomarkers for SMA clinical trials. Neurology 66:1067-73 |
