Biblio
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome. Sci Transl Med. 2024;16(745):eadi8214.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome. N Engl J Med. 2021;385(21):1929-1940.
Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review. Front Immunol. 2023;14:1187959.
Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical management. Eur J Paediatr Neurol. 2024;49:141-154.