Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia

Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia. ClinicalTrials.gov

A Phase 1, Randomized, Double-blind, Placebo-controlled, Multicenter, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Oral VP 20629 in Adult Subjects With Friedreich's Ataxia, Sponsor: ViroPharma

The objectives of the study are:

To evaluate the safety and tolerability of single and multiple oral doses of VP 20629 in subjects with Friedreich's ataxia (FA). [Primary]
To characterize the pharmacokinetics of VP 20629 by investigation of the plasma concentration-time profile following single and multiple oral doses in subjects with FA. [Secondary]
To investigate the pharmacodynamic effects of VP 20629 on plasma 8-isoprostane and malondialdehyde and urinary 8-hydroxydeoxyguanosine concentrations following multiple oral doses in subjects with FA. [Exploratory]

uniQure signs EU commercialization agreement with Chiesi Farmaceutici for first approved gene therapy treatment, and announces EUR 45 million (USD 58 million) in equity and collaboration financing

uniQure signs EU commercialization agreement with Chiesi Farmaceutici for first approved gene therapy treatment, and announces EUR 45 million (USD 58 million) in equity and collaboration financing


Amsterdam, the Netherlands, July 9, 2013

Biochemistry of cardiomyopathy in the mitochondrial disease Friedreich's ataxia

Biochemistry of cardiomyopathy in the mitochondrial disease Friedreich's ataxia. Darius J. R. Lane, Michael Li‑Hsuan Huang, Samantha Ting, Sutharshani Sivagurunathan and Des R. Richardson; Biochem. J. (2013) 453 (321–336) (Printed in Great Britain) doi:10.1042/BJ20130079

Key words: apoptosis, autophagy, cardiomyopathy, frataxin, Friedreich’s ataxia, integrated stress response.