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European Journal of Neurology|May 26, 2023
Long-term follow-up of 64 children with classical infantile-onset Pompe disease since 2004: A French real-life observational studyMarine Tardieu, Céline Cudejko, Aline Cano, et al.Bone Marrow Transplantation|December 9, 2022
Long term follow-up after haematopoietic stem cell transplantation for mucopolysaccharidosis type I-H: a retrospective study of 51 patientsAntoine Gardin, Martin Castelle, Samia Pichard, et al.Journal of Medical Genetics|March 19, 2021
Disentangling molecular and clinical stratification patterns in beta-galactosidase deficiencyAbdellah Tebani, Bénédicte Sudrié-Arnaud, Ivana Dabaj, et al.Pageof 3