Orphanet Journal of Rare Diseases

Orphanet Journal of Rare Diseases

The following new articles have just been published in Orphanet Journal of Rare Diseases For articles using Author Version-first publication you will see a provisional PDF corresponding to the accepted manuscript. In these instances, the fully formatted Final Version PDF and full text (HTML) versions will follow in due course.

Research   Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act Mechler K, Mountford WK, Hoffmann GF, Ries MOrphanet Journal of Rare Diseases 2015, 10 :46 (18 April 2015) Abstract | Full Text | PDF Research   Effect of cyclic, low dose pyrimethamine treatment in patients with Late Onset Tay Sachs: an open label, extended pilot study Osher E, Fattal-Valevski A, Sagie L, Urshanski N, Sagiv N, Peleg L, Lerman-Sagie T, Zimran A, Elstein D, Navon R, Valevski A, Stern NOrphanet Journal of Rare Diseases 2015, 10 :45 (17 April 2015) Abstract | Full Text | PDF