Sunday, July 22, 2012

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

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European Medicines Agency recommends first gene therapy for approval. European Medicines Agency, Press release 20/07/2012   Gene therapy ...
Saturday, July 21, 2012

Gene, Stem Cell, and Future Therapies for Orphan Disease

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State of the Art. Gene, Stem Cell, and Future Therapies for Orphan Disease . M Ian Phillips. Clinical Pharmacology & Therapeutics (2012)...
Friday, July 20, 2012

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

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The central role and mechanisms of β-cell dysfunction and death in Friedreich's ataxia-associated diabetes . Miriam Cnop, Mariana Igoill...

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

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The Role of Academic Institutions in the Development of Drugs for Rare and Neglected Diseases. L D Coles and J C Cloyd. Clinical Pharmacolo...

The Future of Orphan Drug Development

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The Future of Orphan Drug Development . J Woodcock. Clinical Pharmacology & Therapeutics (2012); 92 2, 146–148. doi:10.1038/clpt.2012.89...
Thursday, July 19, 2012

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

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Skeletal Muscle Involvement in Friedreich Ataxia and Potential Effects of Recombinant Human Erythropoietin Administration on Muscle Regenera...

The Advocate is FARA's newsletter, Summer 2012

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The Advocate is FARA's newsletter. It provides updates on FARA's activities including funded research, scientific conferences, and...
Wednesday, July 18, 2012

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

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"Cell-Replacement Therapies For Neurological Conditions Via Neurons Derived From Cord Blood Cells." Medical News Today. MediLexic...

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

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Activating mitochondrial regulator PGC-1α expression by astrocytic NGF is a therapeutic strategy for Huntington's disease . Li-Wen Chen,...
Monday, July 16, 2012

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

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Opportunities in systems biology to discover mechanisms and repurpose drugs for CNS diseases . Hongkang Mei, Tian Xia, Gang Feng, Jason Zhu,...