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.

Opportunities in systems biology to discover mechanisms and repurpose drugs for CNS diseases

Opportunities in systems biology to discover mechanisms and repurpose drugs for CNS diseases. Hongkang Mei, Tian Xia, Gang Feng, Jason Zhu, Simon Lin, Yang Qiu. Drug Discovery Today, Available online 29 June 2012. DOI: http://dx.doi.org/10.1016/j.drudis.2012.06.015

Keywords: Therapies, central nervous system (CNS) diseases, therapeutic targets, deciphering disease mechanisms, Multiple Level Network Modeling (MLNM) analysis.

The biomechanics of the reaching movement in Friedreich and stroke patients

The biomechanics of the reaching movement in Friedreich and stroke patients. M. Caimmi, M. Malosio, N. Pedrocchi, F. Vicentini, L. Molinari Tosatti, F. Molteni. Gait & Posture, Volume 36, Issue null, Page S88. DOI: http://dx.doi.org/10.1016/j.gaitpost.2011.10.331

Tat-Frataxin protects dopaminergic neuronal cells against MPTP-induced toxicity in a mouse model of Parkinson’s disease

Tat-Frataxin protects dopaminergic neuronal cells against MPTP-induced toxicity in a mouse model of Parkinson’s disease. Mi Jin Kim, Dae Won Kim, Hoon Jae Jeong, Eun Jeong Sohn, Min Jea Shin, Eun Hee Ahn, Soon Won Kwon, Young Nam Kim, Duk-Soo Kim, Jinseu Park, Won Sik Eum, Hyun Sook Hwang, Soo Young Choi. Biochimie, , Available online 14 July 2012. http://dx.doi.org/10.1016/j.biochi.2012.07.005

 Keywords: Frataxin, MPTP, Parkinson’s disease, Protein transduction domain, reactive oxygen species, dopaminergic neurons, blood brain barrier (BBB).

Role of mismatch repair enzymes in GAA-TTC triplet-repeat expansion in Friedreich's ataxia induced pluripotent stem cells (iPSCs)

Role of mismatch repair enzymes in GAA-TTC triplet-repeat expansion in Friedreich's ataxia induced pluripotent stem cells (iPSCs) . Jintang Du, Erica Campau, Elisabetta Soragni, Sherman Ku, James W. Puckett, Peter B. Dervan and Joel M. Gottesfeld. J. Biol. Chem. jbc.M112.391961. First Published on July 13, 2012, doi:10.1074/jbc.M112.391961

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Therapeutic Developments in Friedreich Ataxia

Therapeutic Developments in Friedreich Ataxia. Wilson RB, J Child Neurol 2012 Jul 12. doi: 10.1177/0883073812449691.

Keywords: Friedreich ataxia, neuro- and cardiodegenerative disorder, no approved therapy, frataxin, iron, oxidative stress, mitochondrial dysfunction.