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About 1,535 results

ALLMedicine™ Beta-thalassemia Center

Research & Reviews  479 results

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
https://clinicaltrials.gov/ct2/show/NCT04770753

Aug 9th, 2022 - The mitapivat group will include approximately 114 participants. The placebo group will include approximately 57 participants.

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
https://clinicaltrials.gov/ct2/show/NCT04770779

Aug 9th, 2022 - The mitapivat group will include approximately 160 participants. The placebo group will include approximately 80 participants.

Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
https://clinicaltrials.gov/ct2/show/NCT00061568

Aug 4th, 2022 - Nonmyeloablative allogeneic peripheral blood stem cell (PBSC) transplants are currently being investigated in phase I/II trials assessing engraftment, efficacy, and toxicity at a number of transplant centers. Preliminary data have shown a high rat...

Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia
https://clinicaltrials.gov/ct2/show/NCT05357482

Jul 20th, 2022 - Study Description: We propose to add JSP191, an antibody targeting CD117 (c-Kit), to the 03-H-0170 regimen to improve myeloid chimerism, in patients considered at high risk for complications from or ineligible from standard myeloablative HSCT. Giv...

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
https://clinicaltrials.gov/ct2/show/NCT02105766

Jul 20th, 2022 - Our ongoing nonmyeloablative allogeneic peripheral blood stem cell (PBSC) transplant protocol (03-H-0170) for patients with severe sickle cell disease (SCD) and B-thalassemia from HLA-matched family donors has excellent results thus far. Our long ...

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Drugs  1 results see all →

Clinicaltrials.gov  68 results

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
https://clinicaltrials.gov/ct2/show/NCT04770779

Aug 9th, 2022 - The mitapivat group will include approximately 160 participants. The placebo group will include approximately 80 participants.

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
https://clinicaltrials.gov/ct2/show/NCT04770753

Aug 9th, 2022 - The mitapivat group will include approximately 114 participants. The placebo group will include approximately 57 participants.

Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
https://clinicaltrials.gov/ct2/show/NCT00061568

Aug 4th, 2022 - Nonmyeloablative allogeneic peripheral blood stem cell (PBSC) transplants are currently being investigated in phase I/II trials assessing engraftment, efficacy, and toxicity at a number of transplant centers. Preliminary data have shown a high rat...

Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia
https://clinicaltrials.gov/ct2/show/NCT05357482

Jul 20th, 2022 - Study Description: We propose to add JSP191, an antibody targeting CD117 (c-Kit), to the 03-H-0170 regimen to improve myeloid chimerism, in patients considered at high risk for complications from or ineligible from standard myeloablative HSCT. Giv...

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
https://clinicaltrials.gov/ct2/show/NCT02105766

Jul 20th, 2022 - Our ongoing nonmyeloablative allogeneic peripheral blood stem cell (PBSC) transplant protocol (03-H-0170) for patients with severe sickle cell disease (SCD) and B-thalassemia from HLA-matched family donors has excellent results thus far. Our long ...

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News  66 results

New Therapy May Offer 'Functional Cure' for Two Blood Disorders
https://www.medscape.com/viewarticle/975543

Jun 14th, 2022 - A single treatment of an investigational CRISPR/Cas9-based cell therapy could provide durable disease control for nearly all patients with transfusion-dependent beta-thalassemia and sickle cell disease. After one infusion of exagamglogene autotemc...

FDA Committee Unanimous on Two Gene Therapies
https://www.medscape.com/viewarticle/975467

Jun 13th, 2022 - A US Food and Drug Administration advisory committee gave a unanimous thumbs up to two new gene therapies: betibeglogene autotemcel (beti-cel) for the treatment of a blood disorder, transfusion-dependent beta-thalassemia; and elivaldogene autotemc...

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...

Beta-Thalassemia Gene Therapy Achieves Transfusion Independence
https://www.medscape.com/viewarticle/964649

Dec 12th, 2021 - In patients with transfusion-dependent beta-thalassemia, a single gene therapy infusion is capable of yielding durable transfusion independence and substantial improvements in iron overload, an investigator reported at the annual meeting of the Am...

Beta-thalassemia gene therapy achieves lasting transfusion independence
https://www.mdedge.com/hematology-oncology/article/249809/gene-therapy/beta-thalassemia-gene-therapy-achieves-lasting
Andrew Bowser

Dec 11th, 2021 - In patients with transfusion-dependent beta-thalassemia, a single gene therapy infusion is capable of yielding durable transfusion independence and substantial improvements in iron overload, an investigator reported at the annual meeting of the Am.

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