ViroPharma Licenses Rights From Intellect Neurosciences for Product Candidate for Friedreich's Ataxia

EXTON, Pa., Sept. 30, 2011 /PRNewswire via COMTEX/ -- ViroPharma Incorporated (VPHM) today announced the license of worldwide rights from Intellect Neurosciences, Inc. (ILNS) to its clinical stage drug candidate, OX1, being developed for the treatment of Friedreich's Ataxia (FA), a rare, hereditary, progressive neurodegenerative disease. read more ...

Gene therapies advance towards finish line

Nature Reviews Drug Discovery 10, 719-720 (October 2011) | doi:10.1038/nrd3572

Asher Mullard

News and Analysis. Over a decade since gene therapy development came to a near standstill with the death of a clinical trial participant, the field is overcoming issues of immunogenicity, carcinogenicity, manufacturing and small patient populations.....

“I am certain that a gene therapy is going to cross the finish line within the next year or two, in either the United States or the European Union,” says Coté.

Neural Substrates for the Motivational Regulation of Motor Recovery after Spinal-Cord Injury

I am aware that the casuistry of a spinal injury is very distant from the AF, but this paper shows a link between the willingness and recovery progress , or in our case, to preserve the faculties for longer.

PLoS ONE, 2011; 6 (9): e24854 DOI: 10.1371/journal.pone.0024854

Yukio Nishimura1,2,3,7*, Hirotaka Onoe3,4, Kayo Onoe5, Yosuke Morichika1, Hideo Tsukada3,6, Tadashi Isa1,3,7

1 Department of Developmental Physiology, National Institute for Physiological Sciences, Okazaki, Japan, 2 Precursory Research for Embryonic Science and Technology, Japan Science and Technology Agency, Tokyo, Japan, 3 Core Research for Evolutional Science and Technology, Japan Science and Technology Agency, Kawaguchi, Japan, 4 Functional Probe Research Laboratory, RIKEN Center for Molecular Imaging Science, Kobe, Japan, 5 Molecular Dynamics Laboratory, RIKEN Center for Molecular Imaging Science, Kobe, Japan, 6 Central Research Laboratory, Hamamatsu Photonics, Hamamatsu, Japan, 7 Graduate University for Advanced Studies (SOKENDAI), Hayama, Japan

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"It is believed that depression impedes and motivation enhances functional recovery after neuronal damage such as spinal-cord injury and stroke. However, the neuronal substrate underlying such psychological effects on functional recovery remains unclear. "

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In layman's words

ScienceDaily (Sep. 28, 2011) — An effective recovery has been observed in stroke patients and those with spinal cord injuries who have strong vitality and motivation to rehabilitate in clinical practice. However, it was not really clear how motivation facilitates functional recovery in brain science. read more....

Understanding the function of frataxin in eukaryotes

PhD Research Project, Medical Research Council, National Institute for Medical Research, PhD Supervisor: Dr A Pastore
Application Deadline: 30 November 2011

The work should lead both to a better understanding of the mechanisms of iron-sulfur cluster formation and to the establishment of the frataxin function.

Mitochondrial disorders and the eye

Eye and Brain, September 2011 Volume 2011:3 Pages 29 - 47
DOI: http://dx.doi.org/10.2147/EB.S16192

Van Bergen NJ, Chakrabarti R, O’Neill EC, Crowston JG, Trounce IA

Keywords: mitochondria, disease, retina, eye, aging, neuroprotection, Friedreich’s ataxia.

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1st iissue off EFACTS NEWS

The EFACTS (European Friedreich’s Ataxia Consortium for Translational Studies) Consortium will publish the newsletter EFACTS NEWS aimed at communicating the activities of the Network and progress in FRDA research to affected families, the general public, health care professionals and the scientific community. The newsletter will be published annually and the issues will appear on the EFACTS Website

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Mutation in Fe-S scaffold Isu bypasses frataxin deletion

Biochem. J. (2011) Immediate Publication, doi:10.1042/BJ20111637

Heeyong Yoon, Ramesh Golla, Emmanuel Lesuisse, Jayashree Pain, Jason Donald, Elise R. Lyver, Debkumar Pain and Andrew Dancis
University of Pennsylvania, Philadelphia, U.S.A.

Keywords: Frataxin, Friedreich’s ataxia, iron homeostasis, Fe-S cluster assembly, cysteine desulfurase (Nfs1), accessory protein (Isd11), scaffold protein (Isu), single amino acid substitution.

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Orphan Drugs, Big Pharma

Human Gene Therapy. September 2011, 22(9): 1035-1038. doi:10.1089/hum.2011.2515.

—Alex Philippidis, Senior News Editor

Safety and Efficacy of Intravenous Immune Globulin in Treating Friedreich's Ataxia and Spinocerebellar Ataxia

Sponsor: University of South Florida
Collaborator: Baxter Healthcare Corporation
Information provided by (Responsible Party): Theresa Zesiewicz, University of South Florida
ClinicalTrials.gov Identifier: NCT01350440

This study is currently recruiting participants. (Last Updated on September 9, 2011)

Neuroprotective and metabolic effects of resveratrol: Therapeutic implications for huntington's disease and other neurodegenerative disorders

doi:10.1016/j.expneurol.2011.08.014

Giulio Maria Pasinettilow asterisk, a, E-mail The Corresponding Author, Jun Wanga, Philippe Marambauda, Mario Ferruzzia, Paul Gregora, Lindsay Alexis Knablea and Lap Hoa
a Center of Excellence for Research in Complementary and Alternative Medicine in Alzheimer's Disease, Department of Neurology, Mount Sinai School of Medicine, New York, NY 1002

Keywords: Resveratrol, polyphenolic compound, aging, metabolic disorders, inflammation , cancer, currently being tested in numerous clinical trials, key metabolic sensor/effector proteins, potential neuroprotective effects, SRT501 , Huntington's disease (HD)., diabetes mellitus.