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 ...
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 ...
Rarity PBC, a Public Benefit Corporation dedicated to expanding the availability of transformative gene therapies for rare diseases, today announced the closing of a $4.6 million seed financing led by ...
A blood stem cell gene therapy co-developed by UCLA’s Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications ...
Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...
Two months after she was born, Eliana Nachem got a cough that wouldn’t go away. Three weeks later, she also started having runny stool, prompting a visit to her pediatrician. Eliana didn’t have ...
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