Long-term outcomes of early enzyme replacement therapy for mucopolysaccharidosis IV: Clinical case studies of two siblings

Sharon Barak, Yair Anikster, Ifat Sarouk, Eve Stern, Etzyona Eisenstein, Tamar Yissar, Nir Sherr-Lurie, Annick Raas-Rothschild, Dafna Guttman

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Medicine and Dentistry