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Scientific Reports|November 5, 2016
Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patientsElisa Masat, Pascal Laforêt, Marie De Antonio, et al.Science Translational Medicine|November 26, 2025
Base editing of β0-thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathiesGiulia Hardouin, Pierre Martinucci, Samantha Scaramuzza, et al.Pageof 4