Maria Ester Bernardo

How Does Hematopoietic Stem Cell Gene Therapy Affect Hip Dysplasia in Mucopolysaccharidosis Type I Hurler Syndrome? Radiological Outcomes at a Minimum 4-year Follow-up

BACKGROUND: Mucopolysaccharidosis type I (MPS IH), or Hurler syndrome, is a lysosomal disorder caused by glycosaminoglycan accumulation due to an α-L-iduronidase deficiency. It is characterized by progressive skeletal impairment and impaired cognitive development. Hip dysplasia i...