Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Author:

Locatelli Franco1ORCID,Lang Peter1,Wall Donna1,Meisel Roland1,Corbacioglu Selim1ORCID,Li Amanda M.1,de la Fuente Josu1,Shah Ami J.1,Carpenter Ben1,Kwiatkowski Janet L.1,Mapara Markus1,Liem Robert I.1,Cappellini Maria Domenica1,Algeri Mattia1,Kattamis Antonis1,Sheth Sujit1,Grupp Stephan1,Handgretinger Rupert1,Kohli Puja1,Shi Daoyuan1,Ross Leorah1,Bobruff Yael1,Simard Christopher1,Zhang Lanju1,Morrow Phuong Khanh1,Hobbs William E.1,Frangoul Haydar1ORCID

Affiliation:

1. From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) — all in Italy; University Children’s Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) “Image-guided and Functionally Instructed Tumor Therapies” and the German Cancer Consortium, Partner Site Tübingen,...

Funder

CRISPR Therapeutics

Vertex Pharmaceuticals

Publisher

Massachusetts Medical Society

Reference36 articles.

1. Beta-thalassemia

2. β-Thalassemias

3. β-Thalassemia

4. Changes in the quality of life of people with thalassemia major between 2001 and 2009.;Gollo G;Patient Prefer Adherence,2013

5. Patient- and Caregiver-Reported Burden of Transfusion-Dependent β-Thalassemia Measured Using a Digital Application

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