Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, singl... by Kwiatkowski, Janet L, Walters, Mark C, Hongeng, Suradej, Yannaki, Evangelia, Kulozik, Andreas E, Kunz, Joachim B, Sauer, Martin G, Thrasher, Adrian J, Thuret, Isabelle, Lal, Ashutosh, Tao, Ge, Ali, Shamshad, Thakar, Himal L, Elliot, Heidi, Lodaya, Ankit, Lee, Ji, Colvin, Richard A, Locatelli, Franco, Thompson, Alexis A

Get full text

Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion-Dependent β-Thalassemia by Thompson, Alexis, Kwiatkowski, Janet L., Schneiderman, Jennifer, Thuret, Isabelle, Kulozik, Andreas E., Yannaki, Evangelia, Cavazzana, Marina, Hongeng, Suradej, Olson, Timothy S., Sauer, Martin G., Thrasher, Adrian J., Lal, Ashutosh, Rasko, John E. J., Ali, Shamshad, Tao, Ge, Thakar, Himal L., Deora, Ami, Paramore, Clark, Witthuhn, Niki, Walters, Mark C., Locatelli, Franco

Get full text

Improvement in Iron Burden in Patients with Transfusion-Dependent β-Thalassemia (TDT) Treated with Betibeglogene Autotemcel (Beti-cel) Gene Therapy: Up to 9 Years of Follow-up by Kwiatkowski, Janet L., Olson, Timothy S., Walters, Mark C., Porter, John B., Schneiderman, Jennifer, Hongeng, Suradej, Kulozik, Andreas, Cavazzana, Marina, Sauer, Martin G., Thrasher, Adrian J., Thuret, Isabelle, Lal, Ashutosh, Rasko, John E. J., Yannaki, Evangelia, Ali, Shamshad, Tao, Gloria, Deora, Ami, Thakar, Himal L., Colvin, Richard A., Locatelli, Franco, Thompson, Alexis A.

Get full text

Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Au... by Thompson, Alexis A., Olson, Timothy S., Walters, Mark C., Porter, John B., Schneiderman, Jennifer, Hongeng, Suradej, Kulozik, Andreas, Cavazzana, Marina, Sauer, Martin G., Thrasher, Adrian J., Thuret, Isabelle, Lal, Ashutosh, Rasko, John E. J., Yannaki, Evangelia, Ali, Shamshad, Tao, Gloria, Thakar, Himal L., Deora, Ami, Gruppioni, Katiana, Colvin, Richard A., Locatelli, Franco, Kwiatkowski, Janet L.

Get full text

Mobilization and Apheresis Experience in Patients Receiving One-Time Gene Therapy with Beti-Cel for Patients with Transfusion-Dependent β-Thalassemia: A Pooled Analysis by Schneiderman, Jennifer, Friedman, David F., Anderson, Christina, Tao, Ge, Ali, Shamshad, Petrusich, Alexandria, Thakar, Himal L., Colvin, Richard A., Yannaki, Evangelia

Get full text

Sustained Efficacy and Safety in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel) by Walters, Mark C., Thompson, Alexis A., Olson, Timothy S., Porter, John B., Schneiderman, Jennifer, Hongeng, Suradej, Kulozik, Andreas E., Cavazzana, Marina, Sauer, Martin G., Thrasher, Adrian J., Thuret, Isabelle, Lal, Ashutosh, Rasko, John E.J., Yannaki, Evangelia, Ali, Shamshad, Tao, Ge, Thakar, Himal L., Deora, Ami, Gruppioni, Katiana, Colvin, Richard A., Locatelli, Franco, Kwiatkowski, Janet L.

Get full text

Evaluation of Mobilization, Apheresis, and Conditioning Regimen and Engraftment in Patients Receiving One-Time Gene Therapy with Elivaldogene Autotemcel (Eli-cel) for Cerebral Adre... by Duncan, Christine N., Orchard, Paul J., Eichler, Florian S., Kühl, Jörn-Sven, De Oliveira, Satiro N., Thrasher, Adrian J., Chiesa, Robert, Dalle, Jean-Hugues, Shah, Ami J., Locatelli, Franco, Smith, Nicholas J.C., Fernandes, Juliana Folloni, Amartino, Hernán M., Lindemans, Caroline A., Zhao, Jack, Singh, Ajay, Thakar, Himal L., Prasad, Vinod K., Dietz, Andrew C., Williams, David A.

Get full text

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia by Locatelli, Franco, Walters, Mark C., Kwiatkowski, Janet L., Cavazzana, Marina, Hongeng, Suradej, Porter, John B., Thrasher, Adrian J., Kulozik, Andreas E., Thuret, Isabelle, Rasko, John E.J., Yannaki, Evangelia, Sauer, Martin G., Ali, Shamshad, Thakar, Himal, Gruppioni, Katiana, Thompson, Alexis A.

Get full text