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On December 20, the FDA placed a partial hold on bluebird bio’s clinical program for lovotibeglogene autotemcel (lovo-cel) gene therapy, temporarily stopping testing on study volunteers under age 18. This partial hold was a response to the investigation of one adolescent with sickle cell disease who has persistent anemia (not dependent on transfusion) a year and a half following treatment. Read more.
Adults can continue enrollment and treatment in bluebird bio studies. Other clinical research studies of gene therapy for sickle cell disease are continuing.
The clinical research process has many built-in safety precautions, which include the partial hold implemented by the FDA. MARAC will continue to monitor the situation closely on behalf of the individuals and families living with sickle cell disease.
videos & visualsGene Therapy: Your Questions Answeredhttps://www.youtube.com/watch?v=5ChXI6cS...
news & eventsFoundation for Women and Girls ConferenceJoin Foundation for Women & Girls wi...
news & eventsNovel Sickle Cell Drug Causes Radical Results in End of Life PatientsThis week the FDA approved a novel treat...
news & eventsGamida Cell to Present Data from NiCord® Programs at the 2018 BMT Tandem MeetingsGamida Cell, a leading cellular and immu...
news & eventsSickle Cell Groups, Pfizer Work to Bring African-Americans into Clinical TrialsIt’s been nearly half a century since ...
education & researchTreatment for avascular necrosis of bone in people with sickle cell diseaseBACKGROUND: Avascular necrosis of bone ...
news & eventsSancilio Pharmaceuticals Company, Inc. (SPCI) Receives Rare Pediatric Disease Designation From the US Food and Drug ...Sancilio Pharmaceuticals Company, Inc. (...
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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.