1. 'Approved for use in uveitis': drug approval for an orphan disease. (July 2015) Authors: Metzinger, Jamie Lynne; Foster, C Stephen Journal: Expert opinion on orphan drugs Issue: Volume 3:Number 7(2015:Jul.) Page Start: 799 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. 2-Iminobiotin for the treatment of perinatal asphyxia. (December 2013) Authors: Perrone, Serafina; Santacroce, Antonino; Buonocore, Giuseppe Journal: Expert opinion on orphan drugs Issue: Volume 1:Number 11(2013:Nov.) Page Start: 935 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. 2-Iminobiotin for the treatment of perinatal asphyxia. (November 2013) Authors: Perrone, Serafina; Santacroce, Antonino; Buonocore, Giuseppe Journal: Expert opinion on orphan drugs Issue: Volume 1:Number 11(2013:Nov.) Page Start: 935 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. 20-year follow-up study of Danish HHT patients—survival and causes of death. Issue 1 (December 2016) Authors: Kjeldsen, Anette; Aagaard, Katrine; Tørring, Pernille; Möller, Sören; Green, Anders Journal: Orphanet journal of rare diseases Issue: Volume 11:Issue 1(2016) Page Start: 1 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. 3, 4-Diaminopyridine (amifampridine) for the treatment of Lambert–Eaton myasthenic syndrome. (March 2014) Authors: Lindquist, Sabine; Stangel, Martin Journal: Expert opinion on orphan drugs Issue: Volume 2:Number 3(2014:Mar.) Page Start: 293 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
6. 47 patients with FLNA associated periventricular nodular heterotopia. Issue 1 (December 2015) Authors: Lange, Max; Kasper, Burkhard; Bohring, Axel; Rutsch, Frank; Kluger, Gerhard; Hoffjan, Sabine; Spranger, Stephanie; Behnecke, Anne; Ferbert, Andreas; Hahn, Andreas; Oehl-Jaschkowitz, Barbara; Graul-Neumann, Luitgard; Diepold, Katharina; Schreyer, Isolde; Bernhard, Matthias; Mueller, Franziska; Sie... Journal: Orphanet journal of rare diseases Issue: Volume 9:Issue 1(2014) Page Start: 1 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
7. 8th European Conference on Rare Diseases & Orphan Products (ECRD 2016). Issue 1 (November 2016) Authors: Schlander, Michael; Holm, Søren; Nord, Erik; Richardson, Jeff; Garattini, Silvio; Kolominsky-Rabas, Peter; Marshall, Deborah; Persson, Ulf; Postma, Maarten; Simoens, Steven; de Solà Morales, Oriol; Tolley, Keith; Toumi, Mondher; Telser, Harry; Bonham, James; Hintner, Helmut; Diem, Anja; Laimer, M... Journal: Orphanet journal of rare diseases Issue: Volume 11:Issue 1(2016) Page Start: 1 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
8. A better fit? Biotech versus Big Pharma in orphan/rare disease drug research. (April 2014) Authors: O'Neil, Deborah A Journal: Expert opinion on orphan drugs Issue: Volume 2:Number 4(2014:Apr.) Page Start: 317 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
9. A case of fatal Type I congenital disorders of glycosylation (CDG I) associated with low dehydrodolichol diphosphate synthase (DHDDS) activity. Issue 1 (December 2016) Authors: Sabry, S.; Vuillaumier-Barrot, S.; Mintet, E.; Fasseu, M.; Valayannopoulos, V.; Héron, D.; Dorison, N.; Mignot, C.; Seta, N.; Chantret, I.; Dupré, T.; Moore, S. Journal: Orphanet journal of rare diseases Issue: Volume 11:Issue 1(2016) Page Start: 1 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
10. A closer look at nerve growth factor: from biology to clinical trials in ophthalmology. (March 2015) Authors: Mantelli, Flavio; Sacchetti, Marta; Scuderi, Gianluca; Lambiase, Alessandro Journal: Expert opinion on orphan drugs Issue: Volume 3:Number 3(2015:Mar.) Page Start: 329 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗