Publications in collaboration with researchers from Great Ormond Street Hospital (4)

2023

  1. A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B

    Journal of Clinical Investigation, Vol. 133, Núm. 2

  2. A second update on mapping the human genetic architecture of COVID-19

    Nature

  3. Adeno-associated virus 2 infection in children with non-A–E hepatitis

    Nature, Vol. 617, Núm. 7961, pp. 555-563

2022

  1. Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB

    Journal of Pediatrics