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A 13-year-old boy with sickle cell disease has become the world’s first patient to be successfully treated with gene therapy. The unidentified teenager remains free from sickle crises and other signs of the disease 15 months after his treatment, which was conducted as part of a Phase 1/2 clinical trial (NCT02151526).
The research team, led by Marina Cavazzana, MD, PhD, a hematology professor at Paris-Descartes University in France, inserted an antisickling β-globin gene, with the help of a viral carrier, into the boy’s blood-producing stem cells.
The report, “Gene Therapy in a Patient with Sickle Cell Disease,” appeared in the New England Journal of Medicine.
education & researchWarmed Saline for Vaso-Oclusive Episodes in Sickle Cell DiseaseInvestigators from the University of Mar...
news & eventsSCD Patients Benefit From Early Rivipansel Treatment for VOCs, New Analyses ShowStarting treatment with rivipansel (GMI-...
news & eventsImara Reports Favorable Preclinical and Phase 1 Data on IMR-687 in Sickle Cell DiseaseImara Inc. today announced it will repor...
education & researchThe effects of relaxation intervention on pain, stress, and autonomic responses among adults with sickle cell pain i...Pain is the major complication for the a...
news & eventsLa Jolla Pharmaceutical company announces initiation of pivotal clinical study of LJPC-401 in patients with beta tha...La Jolla Pharmaceutical company today an...
Community CenterToday’s Faces of Sickle Cell Disease: Courtney Fitzhugh, M.D.Courtney Fitzhugh first became intereste...
videos & visualsSCDAA Gene Therapy Town Hall – Webinarhttps://www.youtube.com/watch?v=J77Gq9yI...
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.