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Orphanet Journal of Rare Diseases
|
October 8, 2015
Erratum to: Establishing medical plausibility in the context of orphan medicines designation in the European Union
Stelios Tsigkos, Segundo Mariz, Jordi Llinares, et al.
Nature Reviews. Drug Discovery
|
June 2, 2016
Worldwide collaboration for orphan drug designation
Segundo Mariz, James H Reese, Kerstin Westermark, et al.
Frontiers in Oncology
|
January 6, 2022
Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation
Jorn Mulder, Tobias van Rossum, Segundo Mariz, et al.
Drug Discovery Today
|
June 26, 2017
Establishing rarity in the context of orphan medicinal product designation in the European Union
Stelios Tsigkos, Matthias Philipp Hofer, Maria Elzbieta Sheean, et al.
Archives of Disease in Childhood
|
November 2, 2017
Comparative analysis of the scope of European Union paediatric investigation plans with corresponding orphan designations
Andrea Ecker, Segundo Mariz, Frauke Naumann-Winter, et al.
Orphanet Journal of Rare Diseases
|
December 6, 2014
Establishing medical plausibility in the context of orphan medicines designation in the European Union
Stelios Tsigkos, Segundo Mariz, Jordi Llinares, et al.
Orphanet Journal of Rare Diseases
|
January 28, 2014
Use of biomarkers in the context of orphan medicines designation in the European Union
Stelios Tsigkos, Jordi Llinares, Segundo Mariz, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 3, 2024
Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe
Gloria M Palomo, Tomas Pose-Boirazian, Frauke Naumann-Winter, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 5, 2023
The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products
Gloria M Palomo, Tomas Pose-Boirazian, Frauke Naumann-Winter, et al.
Gene Therapy
|
March 14, 2024
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Maria Elisabeth Kalland, Tomas Pose-Boirazian, Gloria Maria Palomo, et al.
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of 3
Search research articles
Search
Showing results (11-20 of 26) with videos related to
Sort By:
Page
of 3
Orphanet Journal of Rare Diseases
|
October 8, 2015
Erratum to: Establishing medical plausibility in the context of orphan medicines designation in the European Union
Stelios Tsigkos, Segundo Mariz, Jordi Llinares, et al.
Nature Reviews. Drug Discovery
|
June 2, 2016
Worldwide collaboration for orphan drug designation
Segundo Mariz, James H Reese, Kerstin Westermark, et al.
Frontiers in Oncology
|
January 6, 2022
Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation
Jorn Mulder, Tobias van Rossum, Segundo Mariz, et al.
Drug Discovery Today
|
June 26, 2017
Establishing rarity in the context of orphan medicinal product designation in the European Union
Stelios Tsigkos, Matthias Philipp Hofer, Maria Elzbieta Sheean, et al.
Archives of Disease in Childhood
|
November 2, 2017
Comparative analysis of the scope of European Union paediatric investigation plans with corresponding orphan designations
Andrea Ecker, Segundo Mariz, Frauke Naumann-Winter, et al.
Orphanet Journal of Rare Diseases
|
December 6, 2014
Establishing medical plausibility in the context of orphan medicines designation in the European Union
Stelios Tsigkos, Segundo Mariz, Jordi Llinares, et al.
Orphanet Journal of Rare Diseases
|
January 28, 2014
Use of biomarkers in the context of orphan medicines designation in the European Union
Stelios Tsigkos, Jordi Llinares, Segundo Mariz, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 3, 2024
Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe
Gloria M Palomo, Tomas Pose-Boirazian, Frauke Naumann-Winter, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 5, 2023
The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products
Gloria M Palomo, Tomas Pose-Boirazian, Frauke Naumann-Winter, et al.
Gene Therapy
|
March 14, 2024
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Maria Elisabeth Kalland, Tomas Pose-Boirazian, Gloria Maria Palomo, et al.
Page
of 3