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Blood Advances
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March 24, 2024
Successes and pitfalls in orphan drug development for sickle cell disease
Enrico Costa, Antonella Isgrò, Mariane de Montalembert, et al.
Recenti Progressi in Medicina
|
September 30, 2015
[Considerations on limits and profits of registries]
Antonio Addis, Enrico Costa, Rossana De Palma, et al.
Drug Discovery Today
|
September 4, 2022
Emergent treatments for β-thalassemia and orphan drug legislations
Enrico Costa, Maria Domenica Cappellini, Stefano Rivella, et al.
Bulletin of the World Health Organization
|
January 2, 2024
Uptake of orphan drugs in the WHO essential medicines lists
Enrico Costa, Lorenzo Moja, Veronika J Wirtz, et al.
Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
|
August 25, 2024
Differential times of submission and approval of CFTR modulators for the treatment of Cystic Fibrosis in the United States and the European Union
Enrico Costa, Silvia Girotti, Clément Mathieu, et al.
Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
|
July 28, 2023
Traits, trends and hits of orphan drug designations in cystic fibrosis
Enrico Costa, Silvia Girotti, Hendrika A van den Ham, et al.
Lancet (London, England)
|
July 25, 2018
Outrageous prices of orphan drugs: a call for collaboration
Lucio Luzzatto, Hanna I Hyry, Arrigo Schieppati, et al.
Drug Discovery Today
|
September 1, 2025
Addressing global regulatory challenges in rare disease drug development
Enrico Costa, Vijayalekshmi Ajith, Ahmed F Al Khaldi, et al.
Frontiers in Medicine
|
April 1, 2025
Medicines not recommended for inclusion in the who essential medicines list: a retrospective observational study
Enrico Costa, Vittorio Del Grosso, Bernadette Cappello, 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 4
Search research articles
Search
Showing results (21-30 of 38) with videos related to
Sort By:
Page
of 4
Blood Advances
|
March 24, 2024
Successes and pitfalls in orphan drug development for sickle cell disease
Enrico Costa, Antonella Isgrò, Mariane de Montalembert, et al.
Recenti Progressi in Medicina
|
September 30, 2015
[Considerations on limits and profits of registries]
Antonio Addis, Enrico Costa, Rossana De Palma, et al.
Drug Discovery Today
|
September 4, 2022
Emergent treatments for β-thalassemia and orphan drug legislations
Enrico Costa, Maria Domenica Cappellini, Stefano Rivella, et al.
Bulletin of the World Health Organization
|
January 2, 2024
Uptake of orphan drugs in the WHO essential medicines lists
Enrico Costa, Lorenzo Moja, Veronika J Wirtz, et al.
Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
|
August 25, 2024
Differential times of submission and approval of CFTR modulators for the treatment of Cystic Fibrosis in the United States and the European Union
Enrico Costa, Silvia Girotti, Clément Mathieu, et al.
Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
|
July 28, 2023
Traits, trends and hits of orphan drug designations in cystic fibrosis
Enrico Costa, Silvia Girotti, Hendrika A van den Ham, et al.
Lancet (London, England)
|
July 25, 2018
Outrageous prices of orphan drugs: a call for collaboration
Lucio Luzzatto, Hanna I Hyry, Arrigo Schieppati, et al.
Drug Discovery Today
|
September 1, 2025
Addressing global regulatory challenges in rare disease drug development
Enrico Costa, Vijayalekshmi Ajith, Ahmed F Al Khaldi, et al.
Frontiers in Medicine
|
April 1, 2025
Medicines not recommended for inclusion in the who essential medicines list: a retrospective observational study
Enrico Costa, Vittorio Del Grosso, Bernadette Cappello, 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 4