The Role of microRNAs in the Biology of Rare Diseases

por Salvatore, Marco, Magrelli, Armando, Taruscio, Domenica
Publicado 2011

CAR-T State of the Art and Future Challenges, A Regulatory Perspective

por Giorgioni, Lorenzo, Ambrosone, Alessandra, Cometa, Maria Francesca, Salvati, Anna Laura, Magrelli, Armando
Publicado 2023

Regulatory Standards in Orphan Medicinal Product Designation in the EU

por Tsigkos, Stelios, Mariz, Segundo, Sheean, Maria Elzbieta, Larsson, Kristina, Magrelli, Armando, Stoyanova-Beninska, Violeta
Publicado 2021

Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspective

por Tomeo, Francesca, Mariz, Segundo, Brunetta, Angelo Loris, Stoyanova‐Beninska, Violeta, Penttila, Karri, Magrelli, Armando
Publicado 2021

Using a stated preference discrete choice experiment to assess societal value from the perspective of patients with rare diseases in Italy

por López-Bastida, Julio, Ramos-Goñi, Juan Manuel, Aranda-Reneo, Isaac, Taruscio, Domenica, Magrelli, Armando, Kanavos, Panos
Publicado 2019

Licensing of Orphan Medicinal Products—Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicine...

por Naumann-Winter, Frauke, Wolter, Franziska, Hermes, Ulrike, Malikova, Eva, Lilienthal, Nils, Meier, Tania, Kalland, Maria Elisabeth, Magrelli, Armando
Publicado 2022

Orphan Medicinal Products for the Treatment of Pancreatic Cancer: Lessons Learned From Two Decades of Orphan Designation

por Mulder, Jorn, van Rossum, Tobias, Mariz, Segundo, Magrelli, Armando, de Boer, Anthonius, Pasmooij, Anna M. G., Stoyanova-Beninska, Violeta
Publicado 2021

Real-World Data and Budget Impact Analysis (BIA): Evaluation of a Targeted Next-Generation Sequencing Diagnostic Approach in Two Orthopedic Rare Diseases

por Pedrini, Elena, Negro, Antonella, Di Brino, Eugenio, Pecoraro, Valentina, Sculco, Camilla, Abelli, Elisabetta, Gnoli, Maria, Magrelli, Armando, Sangiorgi, Luca, Cicchetti, Americo
Publicado 2022

Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit–The EU Regulator's Perspective

por Sheean, Maria E., Naumann-Winter, Frauke, Capovilla, Giuseppe, Kalland, Maria Elisabeth, Malikova, Eva, Mariz, Segundo, Matusevicius, Darius, Nistico, Robert, Schwarzer-Daum, Brigitte, Tsigkos, Stelios, Tzogani, Kyriaki, Larsson, Kristina, Magrelli, Armando, Stoyanova-Beninska, Violeta
Publicado 2021