Systemic administration of a brain-penetrant peptide–iduronidase conjugate results in brain IDUA activity in MPS I mice

CO RR EC TE D P RO OF refer the patients earlier to a reference center to establish the definite diagnosis properly. Premature symptom recognition and diagnosis are vital to achieve a better prognosis in patients with MPS and the broader familiarity with clinical manifestations of this lysosomal disorder is of paramount importance to all health care professionals, leading to earlier diagnosis and initiation of specific treatment (in MPS I, II and VI), thus improving the patient outcomes. Financial support was provided by the Institute of Genetics and Inborn Errors of Metabolism (IGEIM).