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Journal Articles

RP-L301, a Lentiviral-Mediated Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase 2 Clinical Trial Design

Ami J Shah, José Luis López Lorenzo, Rachael F Grace, E J van Beers, M Bartels, Andreas Glenthoej, Susana Navarro, Lucia Llanos, Begoña Pérez de Camino Gaisse, Sol Sanchez, Josune Zubicaray, May Chien, Oscar Quintana Bustamante, M González-Vicent, June Iriondo, Miriam Zeini, Grace Choi, Eileen Nicoletti, Maria Chitty-Lopez, Gayatri R Rao, Juan Bueren, Jonathan D Schwartz, Jose Carlos Segovia, Julián Sevilla
Journal: Blood
Blood (2024) 144 (Supplement 1): 3583.1.
https://doi.org/10.1182/blood-2024-201734
Published: 2024
Journal Articles

Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study

Ami J. Shah, José Luis López Lorenzo, Julián Sevilla, Susana Navarro, Lucía Llanos, Begoña Pérez de Camino Gaisse, Sol Sanchez, Josune Zubicaray, Bert Glader, May Chien, Oscar Quintana Bustamante, Miriam Zeini, Grace Choi, Eileen Nicoletti, Gayatri R. Rao, Maria Grazia Roncarolo, Juan A. Bueren, Jonathan D. Schwartz, José C. Segovia
Journal: Blood
Blood (2022) 140 (Supplement 1): 4902–4903.
https://doi.org/10.1182/blood-2022-170948
Published: 2022
Journal Articles

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients

Ami J Shah, José Luis López Lorenzo, Susana Navarro, Julián Sevilla, Lucía Llanos, Begoña Pérez de Camino Gaisse, Sol Sanchez, Bert Glader, May Chien, Oscar Quintana Bustamante, Brian C Beard, Kenneth M Law, Miriam Zeini, Grace Choi, Eileen Nicoletti, Gayatri R Rao, Maria Grazia Roncarolo, Juan A Bueren, Jonathan D Schwartz, José C Segovia
Journal: Blood
Blood (2021) 138 (Supplement 1): 563.
https://doi.org/10.1182/blood-2021-148161
Published: 2021
Journal Articles

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

José Luis López Lorenzo, MD, Susana Navarro, PhD, Ami J Shah, MD, Maria Grazia Roncarolo, MD, Julián Sevilla, MD PhD, Lucía Llanos, MD PhD, Begoña Pérez Camino de Gaisse, MD, Sol Sanchez, MD, Bertil Glader, MD PhD, May Chien, MD, Oscar Quintana Bustamante, PhD, Brian C Beard, PhD, Kenneth M Law, PhD, Miriam Zeini, PhD, Grace Choi, BS, Eileen Nicoletti, MD, Juan A Bueren, PhD, Gayatri R Rao, MD JD, Jonathan D Schwartz, MD, José C Segovia, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 47.
https://doi.org/10.1182/blood-2020-137246
Published: 2020
Journal Articles

Incidence, Diagnosis, and Outcome of Acquired Thrombotic Thrombocytopenic Purpura (aTTP): A Nationwide Survey By the Spanish Apheresis Group

Maria Cristina Pascual Izquierdo, PhD MD, Julio Del Rio-Garma, MD, Javier De La Rubia, Aurora Viejo, MD, Jesus Fernandez-Sojo, MD, Jesus Martin, MD, Xavier Solanich, MD, Faustino García-Candel, PhD MD, Luis Hernandez, MD, Marta Fernandez, MD, Ana Oliva, MD, Jose Maria Garcia-Gala, MD, Maria Eva Mingot-Castellano, PhD MD, Ines Gomez-Seguí, MD PhD, Rosa Goterris, MD, Michael Calviño, MD, María Luisa Antelo, MD PhD, Sol Sanchez, MD, Jose Antonio Moreno, MD, Cristina Amunarriz, MD, Jose Garcia-Arroba, MD, Carmen Fernandez, MD, Nieves Alonso, MD, Victoria Gonzalez, MD, Luisa Guerra, MD, M. Elena Moreno, MD, Moreno M. Dolores, MD, Gemma Moreno, MD, Melisa Daorta, MD, Esther Chica, MD, Rafael Del Orbe, MD PhD, Joan Cid, MD PhD, Margarita Berberana, MD, Carmen Ballester, MD, Sara Nistal Gil, MD, Julia Vidan, MD, Maite Calderon, MD, Consuelo Martinez Redondo, MD, Jose Carlos Hernandez, MD, Marina Gordillo, MD, Angel Salgado, Ramon Salinas, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4910.
https://doi.org/10.1182/blood-2019-123258
Published: 2019