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About 865 results

ALLMedicine™ Beta Thalassemia Center

Research & Reviews  251 results

Assessment of Cognitive Function, Fatigue and Health Related Quality of Life in Children With Beta Thalassemia
https://clinicaltrials.gov/ct2/show/NCT05469230

Jul 22nd, 2022 - The cognitive abilities are extremely important for academic achievement. Despite their importance, the evaluation of cognitive function is rarely performed for thalassemia patients. So it is important to assess it and should be a routine comprehe...

Influenza Vaccine Booster Stimulates Antibody Response in Beta Thalassemia Major Patients.
https://doi.org/10.1093/labmed/lmac035
Laboratory Medicine; Sheikh M, Ahmadi-Vasmehjani A et. al.

Jul 19th, 2022 - The aim of this study was to evaluate antibody response against influenza vaccine in beta thalassemia major patients from Iran. Thirty beta thalassemia major patients were enrolled and divided into three groups: single dose (group 1), double dose ...

Evaluation of coagulation parameters and impact of transfusion on coagulation in patien...
https://doi.org/10.1097/MBC.0000000000001131
Blood Coagulation & Fibrinolysis : an International Journ... Kurt ZDÇ, Sönmez Ç et. al.

Jul 9th, 2022 - There have been several studies that have shown that patients with beta thalassemia major are at a higher risk of thrombosis due to the procoagulant activity of thalassemic erythrocytes, decreased liver synthetic function, increased platelet activ...

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
https://clinicaltrials.gov/ct2/show/NCT05444894

Jul 6th, 2022 - This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant in adult participants with TDT, age 18 to 35 years,...

A Study of IMR-687 in Subjects With Beta Thalassemia
https://clinicaltrials.gov/ct2/show/NCT04411082

Jun 30th, 2022 - A phase 2, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of IMR-687 (phosphodiesterase (PDE) 9 inhibitor) administered once daily (qd) orally for 36 weeks in 2 populations of adult subjects wit...

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Drugs  1 results see all →

Clinicaltrials.gov  56 results

Assessment of Cognitive Function, Fatigue and Health Related Quality of Life in Children With Beta Thalassemia
https://clinicaltrials.gov/ct2/show/NCT05469230

Jul 22nd, 2022 - The cognitive abilities are extremely important for academic achievement. Despite their importance, the evaluation of cognitive function is rarely performed for thalassemia patients. So it is important to assess it and should be a routine comprehe...

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
https://clinicaltrials.gov/ct2/show/NCT05444894

Jul 6th, 2022 - This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant in adult participants with TDT, age 18 to 35 years,...

A Study of IMR-687 in Subjects With Beta Thalassemia
https://clinicaltrials.gov/ct2/show/NCT04411082

Jun 30th, 2022 - A phase 2, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of IMR-687 (phosphodiesterase (PDE) 9 inhibitor) administered once daily (qd) orally for 36 weeks in 2 populations of adult subjects wit...

Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
https://clinicaltrials.gov/ct2/show/NCT02675959

Jun 21st, 2022 - This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem ce...

Thrombophilia In Beta Thalassemia
https://clinicaltrials.gov/ct2/show/NCT04219449

May 17th, 2022 - β-thalassemia represents a major public health problem in Egypt, it is estimated that there are 1000/1.5 million per year live births born with β-thalassemia. The average life expectancy of patients with β-thalassemia has improved over the last fe...

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News  36 results

Gene Therapy for Beta Thalassemia Safe, Effective Up to 8 Years, but Concerns Emerge
https://www.medscape.com/viewarticle/966016

Jan 5th, 2022 - NEW YORK (Reuters Health) - Lentiviral globin gene therapy with reduced-intensity conditioning (RIC) was safe and effective in beta-thalassemia in a 6-8-year follow-up, although cautious monitoring is still necessary, researchers say. Beta-thalass...

FDA Grants Priority Review to Luspatercept-aamt for Non–Transfusion Dependent Beta Thalassemia
https://www.onclive.com/view/fda-grants-priority-review-to-luspatercept-aamt-for-non-transfusion-dependent-beta-thalassemia

Dec 3rd, 2021 - The FDA has granted priority review to the supplemental biologics license application (sBLA) for luspatercept-aamt (Reblozyl) for the treatment of anemia in adults with non–transfusion dependent (NTD) β-thalassemia. The European Medicines Agency h...

Luspatercept Benefit Also in Non-Transfusion-Dependent Thalassemia
https://www.medscape.com/viewarticle/952998

Jun 13th, 2021 - The first-in-class erythroid maturation agent luspatercept (Reblozyl) is already approved for the treatment of anemia in adult patients with beta thalassemia who require red blood cell (RBC) transfusions. Now it has also shown to be beneficial for...

Negative Effects Associated with Iron Overload
https://www.onclive.com/view/negative-effects-associated-with-iron-overload

Apr 2nd, 2021 - Transcript:David P. Steensma, MD: In patients with congenital anemias, like severe beta thalassemia, it’s clear that iron is a major contributor to morbidity and mortality. If you don’t adequately chelate patients with severe transfusion-defendant...

FDA Approvals in MCL and Beta Thalessemia-Associated Anemia, Priority Review in HCC, and More
https://www.onclive.com/view/fda-approvals-in-mcl-and-beta-thalessemia-associated-anemia-priority-review-in-hcc-and-more

Apr 2nd, 2021 - Today- FDA approvals in mantle cell lymphoma and beta thalassemia—associated anemia, a priority review designation in hepatocellular carcinoma, encouraging findings in graft-versus-host disease, and disappointing findings in a biliary tract can...

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Patient Education  2 results see all →