Biblio
Access to gene therapy for rare diseases when commercialization is not fit for purpose. Nat Med. 2023.
. Severe combined immunodeficiency: improved survival leading to detection of underlying liver disease. BMC Gastroenterol. 2023;23(1):166.
. [Adenosine deaminase 2 deficiency: a disease with multiple presentations]. Rev Med Suisse. 2022;18(776):669-673.
. Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry. J Clin Immunol. 2020.
. Busulfan Pharmacokinetics in ADA SCID Gene Therapy. Biol Blood Marrow Transplant. 2020.