1. Comparative analysis of the scope of European Union paediatric investigation plans with corresponding orphan designations. Issue 5 (31st October 2017) Authors: Ecker, Andrea; Mariz, Segundo; Naumann-Winter, Frauke; Norga, Koenraad; Barisic, Ingeborg; Girard, Thomas; Tomasi, Paolo; Mentzer, Dirk; Sepodes, Bruno Journal: Archives of disease in childhood Issue: Volume 103:Issue 5(2018) Page Start: 427 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Designation of orphan conditions in Europe: regulatory observations and considerations after implementation of regulation 141/2000. (2nd June 2020) Authors: Mariz, Segundo; Westermark, Kerstin; Sepodes, Bruno Journal: Expert opinion on orphan drugs Issue: Volume 8:Number 6(2020) Page Start: 189 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Erratum to: Establishing medical plausibility in the context of orphan medicines designation in the European Union. Issue 1 (December 2015) Authors: Tsigkos, Stelios; Mariz, Segundo; Llinares, Jordi; Fregonese, Laura; Aarum, Stiina; Naumann-Winter, Frauke; Westermark, Kerstin; Sepodes, Bruno 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) ↗
4. Establishing rarity in the context of orphan medicinal product designation in the European Union. Issue 3 (March 2018) Authors: Tsigkos, Stelios; Hofer, Matthias Philipp; Sheean, Maria Elzbieta; Mariz, Segundo; Larsson, Kristina; Naumann-Winter, Frauke; Fregonese, Laura; Sepodes, Bruno Journal: Drug discovery today Issue: Volume 23:Issue 3(2018) Page Start: 681 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. EU regulations. (11th January 2011) Authors: Mariz, Segundo; Llinares, Jordi; Westermark, Kerstin Journal: BMJ Issue: Volume 342(2011) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
6. Evolving prevalence of haematological malignancies in orphan designation procedures in the European Union. Issue 1 (December 2017) Authors: Polsinelli, Benedetta; Tsigkos, Stelios; Naumann-Winter, Frauke; Mariz, Segundo; Sepodes, Bruno Journal: Orphanet journal of rare diseases Issue: Volume 12:Issue 1(2017) Page Start: 1 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
7. Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspective. Issue 11 (12th April 2021) Authors: Tomeo, Francesca; Mariz, Segundo; Brunetta, Angelo Loris; Stoyanova‐Beninska, Violeta; Penttila, Karri; Magrelli, Armando Journal: British journal of clinical pharmacology Issue: Volume 87:Issue 11(2021) Page Start: 4183 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
8. Nonclinical data supporting orphan medicinal product designations in the area of rare infectious diseases. Issue 2 (February 2020) Authors: Sheean, Maria E.; Malikova, Eva; Duarte, Dinah; Capovilla, Giuseppe; Fregonese, Laura; Hofer, Matthias P.; Magrelli, Armando; Mariz, Segundo; Mendez-Hermida, Fernando; Nistico, Robert; Leest, Tim; Sipsas, Nikolaos V.; Tsigkos, Stelios; Vitezic, Dinko; Larsson, Kristina; Sepodes, Bruno; Stoyanova-... Journal: Drug discovery today Issue: Volume 25:Issue 2(2020) Page Start: 274 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
9. Nonclinical data supporting orphan medicinal product designations: lessons from rare neurological conditions. Issue 1 (January 2018) Authors: Sheean, Maria E.; Stoyanova-Beninska, Violeta; Capovilla, Giuseppe; Duarte, Dinah; Hofer, Matthias P.; Hoffmann, Michel; Magrelli, Armando; Mariz, Segundo; Tsigkos, Stelios; Shaili, Evyenia; Polsinelli, Benedetta; Ricciardi, Mario; Bonelli, Milton; Balabanov, Pavel; Larsson, Kristina; Sepodes, Bruno Journal: Drug discovery today Issue: Volume 23:Issue 1(2018) Page Start: 26 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
10. The orphan framework as a new opportunity: an expert opinion. (November 2014) Authors: Mariz, Segundo; Tsigkos, Stelios; Fregonese, Laura; Aarum, Stiina; Dehlink, Eleonora; Llinares, Jordi; Sepodes, Bruno Journal: Expert opinion on orphan drugs Issue: Volume 2:Number 11(2014:Nov.) Page Start: 1181 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗