Adenosine deaminase (ADA) deficiency is a rare autosomal recessive disorder that underpins a severe form of combined immunodeficiency (SCID), resulting in the accumulation of toxic metabolites that ...
MILAN, BOSTON, and LONDON, Sept. 12, 2023 /PRNewswire/ -- Fondazione Telethon, one of the main Italian biomedical charities, and Orchard Therapeutics, a global gene therapy leader, today announced the ...
Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy. HealthDay News — Autologous CD34+ hematopoietic stem-cell ...
Following a positive opinion from the EMA, the transfer of marketing authorization of Strimvelis from Orchard Therapeutics has been approved by the European Commission Fondazione Telethon will now be ...
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