Role of resveratrol and its analogues in the treatment of neurodegenerative diseases: focus on recent discoveries.

Role of resveratrol and its analogues in the treatment of neurodegenerative diseases: focus on recent discoveries.V. Foti Cuzzola, R. Ciurleo, S. Giacoppo, S. Marino, P. Bramanti. CNS Neurol Disord Drug Targets. 2011 Nov;10(7):849-62. Keywords: Neurodegenerative diseases, inflammatory mechanism, blood-brain barrier, Resveratrol (RSV), cardio- and neuro-protection, immune regulation, cancer chemoprevention, tumor necrosis factor, cyclooxygenase 2, inducible nitric oxide synthase, interleukins.

European Medicines Agency gives first approval of gene therapy for rare disease

European Medicines Agency recommends first gene therapy for approval. European Medicines Agency, Press release 20/07/2012  

Gene therapy is a great hope for rare diseases of genetic origin, such as Friedreich's ataxia. This is a big step, gene therapy can come out the research laboratories and may be sold by a pharmaceutical laboratory, this authorization would boost the interest of pharmaceutical companies to invest in the development of these therapies.

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Gene, Stem Cell, and Future Therapies for Orphan Disease

State of the Art. Gene, Stem Cell, and Future Therapies for Orphan Disease. M Ian Phillips. Clinical Pharmacology & Therapeutics (2012); 92 2, 182–192. doi:10.1038/clpt.2012.82

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The central role and mechanisms of β-cell dysfunction and death in Friedreich's ataxia-associated diabetes

The central role and mechanisms of β-cell dysfunction and death in Friedreich's ataxia-associated diabetes. Miriam Cnop, Mariana Igoillo-Esteve, Myriam Rai, Audrey Begu, Yasmina Serroukh, Chantal Depondt, Anyishai E Musuaya, Ihsane Marhfour, Laurence Ladrière, Xavier Moles Lopez, Dionysios Lefkaditis, Fabrice Moore, Jean-Pierre Brion, J Mark Cooper, Anthony HV Schapira, Anne Clark, Arnulf H Koeppen, Piero Marchetti, Massimo Pandolfo, Décio L Eizirik and Françoise Féry.  Annals of Neurology. Accepted manuscript online: 20 JUL 2012. DOI: 10.1002/ana.23698

Keywords: Friedreich's ataxia (FRDA), neurodegenerative disease, frataxin gene, diabetes, insulin resistance, β-cell failure, disposition index, Pancreatic β-cell dysfunction, mitochondrial dysfunction.

The Role of Academic Institutions in the Development of Drugs for Rare and Neglected Diseases

The Role of Academic Institutions in the Development of Drugs for Rare and Neglected Diseases. L D Coles and J C Cloyd. Clinical Pharmacology & Therapeutics (2012); 92 2, 193–202. doi:10.1038/clpt.2012.83.

The Future of Orphan Drug Development

The Future of Orphan Drug Development. J Woodcock. Clinical Pharmacology & Therapeutics (2012); 92 2, 146–148. doi:10.1038/clpt.2012.89

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Skeletal Muscle Involvement in Friedreich Ataxia and Potential Effects of Recombinant Human Erythropoietin Administration on Muscle Regeneration and Neovascularization

Skeletal Muscle Involvement in Friedreich Ataxia and Potential Effects of Recombinant Human Erythropoietin Administration on Muscle Regeneration and Neovascularization. Nachbauer W, Boesch S, Reindl M, Eigentler A, Hufler K, Poewe W, Löscher W, Wanschitz J.;J Neuropathol Exp Neurol. 2012 Jul 16.

Keywords: Friedreich ataxia (FRDA), frataxin, cardiac muscle involvement, mitochondrial dysfunction, skeletal muscle, recombinant human erythropoietin (rhuEPO), neurogenic and myopathic changes.

The Advocate is FARA's newsletter, Summer 2012

The Advocate is FARA's newsletter. It provides updates on FARA's activities including funded research, scientific conferences, and fundraising events

Summer 2012

Cell-Replacement Therapies For Neurological Conditions Via Neurons Derived From Cord Blood Cells

"Cell-Replacement Therapies For Neurological Conditions Via Neurons Derived From Cord Blood Cells." Medical News Today. MediLexicon, Intl., 18 Jul. 2012. Web.

Keywords: transcription factor, cord blood (CB) cells, neuron-like cells, neurological conditions, mesoderm, Sox2, future cell-replacement therapies.

Activating mitochondrial regulator PGC-1α expression by astrocytic NGF is a therapeutic strategy for Huntington's disease

Activating mitochondrial regulator PGC-1α expression by astrocytic NGF is a therapeutic strategy for Huntington's disease. Li-Wen Chen, Lin-Yea Horng, Chia-Ling Wu, Hui-Ching Sung, Rong-Tsun Wu. Neuropharmacology, Volume 63, Issue 4, September 2012, Pages 719-732. DOI: http://dx.doi.org/10.1016/j.neuropharm.2012.05.019

Keywords: Mitochondrial dysfunction, Huntington's disease (HD), NGF gene delivery, brain energy metabolism, neuroprotective effects, peroxisome proliferator-activated receptor gamma coactivator 1-α (PGC-1α), blood–brain barrier, astrocytic NGF inducer, Ganoderma lucidum (GaLu) extract.