Deferiprone for the treatment of Friedreich's ataxia. Massimo Pandolfo1, Laura Hausmann. J. Neurochem.(2013) 126 (Suppl. 1), 142–146. DOI: 10.1111/jnc.12300
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Wednesday, July 17, 2013
Co-enzyme Q10 and idebenone use in Friedreich's ataxia
Co-enzyme Q10 and idebenone use in Friedreich's ataxia. Michael H Parkinson, Jörg B. Schulz, Paola Giunti1; J. Neurochem.(2013) 126 (Suppl. 1), 125–141. DOI: 10.1111/jnc.12322
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Diabetes in Friedreich Ataxia
Diabetes in Friedreich Ataxia. Miriam Cnop, Hindrik Mulder, Mariana Igoillo-Esteve. J. Neurochem.(2013) 126 (Suppl. 1), 94–102. DOI: 10.1111/jnc.12216
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Monitoring progression in Friedreich ataxia (FRDA): the use of clinical scales
Monitoring progression in Friedreich ataxia (FRDA): the use of clinical scales; Katrin Bürk Stefanie R. Schulz, Jörg B. Schulz; J. Neurochem.(2013) 126 (Suppl. 1),118–124. DOI: 10.1111/jnc.12318
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Mitochondrial pathophysiology in Friedreich's ataxia
Mitochondrial pathophysiology in Friedreich's ataxia Pilar González-Cabo and Francesc Palau; J. Neurochem.(2013) 126 (Suppl. 1), 53–64. DOI: 10.1111/jnc.12303
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Monday, July 15, 2013
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]
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
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.
Key words: apoptosis, autophagy, cardiomyopathy, frataxin, Friedreich’s ataxia, integrated stress response.
Saturday, July 13, 2013
Low-cost evaluation and real-time feedback of static and dynamic weight bearing asymmetry in patients undergoing in-patient physiotherapy rehabilitation for neurological conditions
Low-cost evaluation and real-time feedback of static and dynamic weight bearing asymmetry in patients undergoing in-patient physiotherapy rehabilitation for neurological conditions. Joanna Foo, Kade Paterson, Gavin Williams and Ross Clark; Journal of NeuroEngineering and Rehabilitation 2013, 10:74 doi:10.1186/1743-0003-10-74
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Weight bearing asymmetry is common in patients with neurological conditions, and recent advances in gaming technology have produced force platforms that are suitable for use in a clinical setting. The aim of this research is to determine whether commercially-available Wii Balance Boards with customized software providing real-time feedback could be used in a clinical setting to evaluate and improve weight-bearing asymmetry in people with various neurological conditions.
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Weight bearing asymmetry is common in patients with neurological conditions, and recent advances in gaming technology have produced force platforms that are suitable for use in a clinical setting. The aim of this research is to determine whether commercially-available Wii Balance Boards with customized software providing real-time feedback could be used in a clinical setting to evaluate and improve weight-bearing asymmetry in people with various neurological conditions.
Thursday, July 11, 2013
Five markers useful for the distinction of canine mammary malignancy
Five markers useful for the distinction of canine mammary malignancy. Karol M Pawlowski, Henryk Maciejewski, Kinga Majchrzak, Izabella Dolka, Jan A Mol, Tomasz Motyl, Magdalena Król. BMC Veterinary Research 2013, 9:138 doi:10.1186/1746-6148-9-138.
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The other selected gene is mipep which is involved in cellular metabolism. The product of this gene performs the final step in processing of nuclear-encoded proteins targeted to the mitochondrial matrix or inner membrane [17]. This gene may contribute to the functional effects of frataxin deficiency and the clinical manifestations of Friedreich ataxia (FA). The exact function of frataxin is still unclear, however it has been implicated in iron homeostasis, protection from oxidative stress and apoptosis [18]. Conflicting results have been reported regarding the role of frataxin in cellular growth: both frataxin knockdown and frataxin overexpression impair cell growth. Despite the role of frataxin in cancer is unknown, the relation between FA and malignancy has been suggested [19].
OPEN ACCESS
The other selected gene is mipep which is involved in cellular metabolism. The product of this gene performs the final step in processing of nuclear-encoded proteins targeted to the mitochondrial matrix or inner membrane [17]. This gene may contribute to the functional effects of frataxin deficiency and the clinical manifestations of Friedreich ataxia (FA). The exact function of frataxin is still unclear, however it has been implicated in iron homeostasis, protection from oxidative stress and apoptosis [18]. Conflicting results have been reported regarding the role of frataxin in cellular growth: both frataxin knockdown and frataxin overexpression impair cell growth. Despite the role of frataxin in cancer is unknown, the relation between FA and malignancy has been suggested [19].
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