Publicacións en colaboración con investigadores/as de Great Ormond Street Hospital (3)

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. 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