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bluebird bio Announces Publication of Case Study on First Patient with Severe Sickle Cell Disease Treated with Gene Therapy in The New England Journal of Medicine
Patient treated with LentiGlobinTM drug product demonstrates high levels (~50% of total hemoglobin) of anti-sickling hemoglobin (HbAT87Q) 15 months after treatment.
bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, today announced the publication in the New England Journal of Medicine of a case study on Patient 1204, the first patient with severe sickle cell disease (SCD) to be treated with gene therapy. This patient, who was 13 years old at the time of treatment, was treated with LentiGlobin drug product in the HGB-205 clinical study conducted in Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France. The data in the publication reflect 15 months of follow-up, and a brief summary of this patient’s outcomes with 21 months of follow-up was presented at the 58th American Society of Hematology Annual Meeting in December 2016.
videos & visualsDiscovery First in Human: Bringing Awareness of Sickle Cell Diseasehttps://www.youtube.com/watch?v=kffu_YvJ...
education & researchHyperhemolysis syndrome in patients with sickle cell anemia: report of three casesBACKGROUND: Sickle cell anemia (SCA) is ...
news & eventsConversation With Women in the Sickle Cell CommunitySickle Cell Project of NW Ohio at Neighb...
education & researchRandomized Feasibility Trial to Improve Hydroxyurea Adherence in Youth Ages 10–18 Years Through Community Health W...Introduction: The main therapeutic int...
news & eventsSolution to 50-year-old mystery could lead to gene therapy for common blood disordersIn a landmark study that could lead to n...
news & events100 Years But Only One Drug: Sickle Cell Patients Wait For HelpLast week, 100,000 Americans with sickle...
news & eventsSiklos (hydroxyurea) now available through ProCare Pharmacy Care’s Siklos At Home programMedunik USA announced today that...
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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.