Sunday, May 10, 2015

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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