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Monoclonal Gammopathy of Undetermined Significance - Patient Characteristics and Referral Patterns
ConclusionMGUS is often incidentally detected as part of a work up for other medical conditions, and our results reveal that there is a variety of reasons for which monoclonal testing is performed. With recent developments in our understanding of the significance of monoclonal gammopathy and its association with certain renal and organ damage (Fermand et al., 2018; Leung et al., 2012), there may be a change in how the paraproteinemia investigations are utilized by clinicians in different disciplines. It will be important to recognize and establish appropriate indications for testing. Furthermore, MGUS patients present with...
Source: Blood - November 21, 2018 Category: Hematology Authors: Lee, H., Street, L., Tay, J., Grossman, J., Thaell, J. F., Goodyear, D., McCulloch, S., Duggan, P., Neri, P., Jimenez-Zepeda, V. Tags: 651. Myeloma: Biology and Pathophysiology, excluding Therapy: Poster III Source Type: research

Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study
Backgroundβ-globin gene transfer has the potential for substantial clinical benefit in patients with sickle cell disease (SCD). LentiGlobin Drug Product (DP) contains autologous CD34+ hematopoietic stem cells (HSCs) transduced with the BB305 lentiviral vector (LVV), encoding β-globin with an anti-sickling substitution (T87Q). The safety and efficacy of LentiGlobin gene therapy is being evaluated in the ongoing Phase 1 HGB-206 study (NCT02140554). Results in the initial 7 patients treated with LentiGlobin DP from steady state bone marrow harvested (BMH) HSCs using original DP manufacturing process (Group A) demons...
Source: Blood - November 21, 2018 Category: Hematology Authors: Tisdale, J. F., Kanter, J., Mapara, M. Y., Kwiatkowski, J. L., Krishnamurti, L., Schmidt, M., Miller, A. L., Pierciey, F. J., Shi, W., Ribeil, J.-A., Asmal, M., Thompson, A. A., Walters, M. C. Tags: 801. Gene Therapy and Transfer: Gene Therapy for Blood Cell Disorders Source Type: research