Long-Term Neurocognitive Function of Pediatric Patients with Severe Combined Immune Deficiency (SCID): Pre- and Post-Hematopoietic Stem Cell Transplant (HSCT)

por Lin, Malinda, Epport, Karen, Azen, Colleen, Parkman, Robertson, Kohn, Donald B., Shah, Ami J.
Publicado 2008

Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency (PKD)?: Toward an expanded definition of severe PKD

por Schwartz, Jonathan D., Barcellini, Wilma, Grace, Rachel F., Bianchi, Paola, Zanella, Alberto, López Lorenzo, José Luis, Sevilla, Julián, Shah, Ami J., Glader, Bertil, Nicoletti, Eileen, Navarro Ordoñez, Susana, Segovia, José Carlos
Publicado 2022

S270: TRANSFUSION INDEPENDENCE AFTER EXAGAMGLOGENE AUTOTEMCEL IN PATIENTS WITH TRANSFUSION-DEPENDENT ΒETA-THALASSEMIA

por Locatelli, Franco, Lang, Peter, Corbacioglu, Selim, LI, Amanda, De La Fuente, Josu, Wall, Donna, Meisel, Roland, Shah, Ami J., Liem, Robert, Mapara, Markus, Carpenter, Ben, Kwiatkowski, Janet, Domenica Cappellini, Maria, Kattamis, Antonis, Sheth, Sujit, Grupp, Stephan, Kohli, Puja, Shi, Daoyuan, Ross, Leorah, Bobruff, Yael, Simard, Christopher, Zhang, Lanju, Khanh Morrow, Phuong, Hobbs, Bill, Frangoul, Haydar
Publicado 2023