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 ...
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 ...
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 ...
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