Alessandro Aiuti

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...

Work participation in inborn errors of immunity: The dual burden on patients and caregivers

Despite improved survival, occupational challenges in with inborn errors of immunity (IEI) patients and their families remain unmeasured. We conducted a nationwide cross-sectional survey promoted by AIP and ImmunITA, including 420 IEI adults and 110 caregivers, integrating occupa...

The withdrawal of beti-cel therapy for β-Thalassemia from the European market: a multistakeholder perspective

Abstract This report aims to outline the complex and multifaceted framework within which the journey to European market access for a β-thalassemia gene therapy has unfolded, highlighting the clinical, ethical, economic, and regulatory challenges faced by all stakeholders involved...