Gene therapy possibilities for certain heart diseases

Gene therapy possibilities for certain heart diseases. Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University. By bioethicsnewsbot | May 13, 2015

Pill of super-protective 'heavy' fat may be key to eternal youth

Pill of super-protective 'heavy' fat may be key to eternal youth. 13 May 2015 by Jessica Hamzelou, New Scientist, Issue 3021

Mikhail Shchepinov, director of Retrotope, a biotech company based in Los Altos, California, wants eventually to slow down the ageing process. But he is starting with a related problem – treating the inherited movement disorder Friedreich's ataxia, with which ageing shares a mechanism.

The trial launching in June is a safety study. The team will be checking that the doses of heavy fat are well tolerated by 18 people with Friedreich's ataxia.

Metabolomics in Precision Medicine

Magazine Preview: Metabolomics in Precision Medicine. Ian Clift, Ph.D.; From May 15 Issue:Genetic Engineering & Biotechnology News

Dr. Blair studies Friedreich’s ataxia, a disease caused by a mutation in the gene frataxin or by a triplet GAA expansion in intron 1 of the gene

Ageing, neuroinflammation and neurodegeneration

Ageing, neuroinflammation and neurodegeneration. Roberta J Ward, David T. Dexter, Robert R. Crichton; Frontiers in Bioscience, Scholar, 7, 189-204, June 1, 2015

Access to new medicines in Europe: technical review of policy initiatives and opportunities for collaboration and research

Access to new medicines in Europe: technical review of policy initiatives and opportunities for collaboration and research. WHO Regional Office for Europe

English (PDF, 3.1 MB)

Oxidative Stress and the Homeodynamics of Iron Metabolism

Oxidative Stress and the Homeodynamics of Iron Metabolism. Nikolaus Bresgen and Peter M. Eck; Biomolecules 2015, 5(2), 808-847; doi:10.3390/biom5020808

Open access

Src inhibitors modulate frataxin protein levels

Src inhibitors modulate frataxin protein levels. Fabio Cherubini, Dario Serio, Ilaria Guccini, Silvia Fortuni, Gaetano Arcuri, Ivano Condò, Alessandra Rufini, Shadman Moiz, Serena Camerini, Marco Crescenzi, Roberto Testi and Florence Malisan; Hum. Mol. Genet. (2015) doi: 10.1093/hmg/ddv162, First published online: May 6, 2015

Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act

Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act. Konstantin Mechler, William K Mountford, Georg F Hoffmann and Markus Ries; Orphanet Journal of Rare Diseases 2015, 10:46 doi:10.1186/s13023-015-0262-5

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The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development

The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development. Emma Heslop, Cristina Csimma, Volker Straub, John McCall, Kanneboyina Nagaraju, Kathryn R Wagner, Didier Caizergues, Rudolf Korinthenberg, Kevin M Flanigan, Petra Kaufmann, Elizabeth McNeil, Jerry Mendell, Sharon Hesterlee, Dominic J Wells and Kate Bushby; Orphanet Journal of Rare Diseases 2015, 10:49 doi:10.1186/s13023-015-0258-1

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Principles for consistent value assessment and sustainable funding of orphan drugs in Europe

Principles for consistent value assessment and sustainable funding of orphan drugs in Europe. Laura Gutierrez, Julien Patris, Adam Hutchings and Warren Cowell; Orphanet Journal of Rare Diseases 2015, 10:53 doi:10.1186/s13023-015-0269-y

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