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About 16,868 results

ALLMedicine™ Thalassemia Center

Research & Reviews  8,217 results

Surrogate Markers of Subclinical Atherosclerosis and Its Associated Factors in Patients...
https://doi.org/10.1080/03630269.2021.1907405
Hemoglobin Soltani D, Fakhrzadeh H et. al.

Apr 8th, 2021 - β-Thalassemia major (β-TM) is a severe genetic hemoglobin (Hb) disorder with cardiovascular complications such as atherosclerosis due to transfusion-dependent iron overload. We aimed to determine the associated factors with surrogate markers of su...

Hb Bart's Hydrops Fetalis Syndrome and Hb H Disease Caused by Deletional Chiang Rai (- ...
https://doi.org/10.1080/03630269.2021.1906269
Hemoglobin Ruengdit C, Khamphikham P et. al.

Apr 6th, 2021 - α0-Thalassemia (α0-thal) Chiang Rai (- -CR; NC_000016.10: g.144215_188843del) was identified as a novel 44.6 kb deletional type of α-thalassemia (α-thal), removing all α-like globin genes. However, little is known about the deleterious effects of ...

The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-th...
https://doi.org/10.1172/JCI144206
The Journal of Clinical Investigation; Matte A, Federti E et. al.

Apr 6th, 2021 - Anemia in β-thalassemia is related to ineffective erythropoiesis and reduced red cell survival. Excess free heme and accumulation of unpaired α-globin chains impose substantial oxidative stress on β-thalassemic erythroblasts and erythrocytes, impa...

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Guidelines  17 results

bluebird bio Receives Positive Opinion from CHMP for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalas...
http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-receives-positive-opinion-chmp-zynteglotm

Mar 28th, 2019 - bluebird bio, Inc. (Nasdaq:BLUE) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending conditional marketing authorization for ZYNTEGLO™ (autol...

bluebird bio’s LentiGlobin™ Gene Therapy Granted Accelerated Assessment by European Medicines Agency for the Treatment of Transfusion-Dependent β-Thalassemia
http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bios-lentiglobintm-gene-therapy-granted-accelerated

Jul 25th, 2018 - bluebird bio, Inc. (Nasdaq: BLUE) today announced that its investigational LentiGlobin™ gene therapy for the treatment of adolescent and adult patients with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype, was granted an acceler...

Red blood cell specifications for patients with hemoglobinopathies: a systematic review...
https://doi.org/10.1111/trf.14611
Transfusion Compernolle V, Chou ST et. al.

Apr 26th, 2018 - Red blood cell (RBC) transfusions remain essential in the treatment of patients with sickle cell disease (SCD) and β-thalassemia. Alloimmunization, a well-documented complication of transfusion, increases the risk of delayed hemolytic transfusion ...

ASH Policy Statement in Support of Patient Access to Safe and Effective Drugs
http://www.hematology.org/Advocacy/Statements/2642.aspx
American Society of Hematology

Jan 5th, 2017 - As an organization of physicians and scientists who care for desperately ill patients, including those with blood cancers such as leukemia, lymphoma, and myeloma, as well as those with non-malignant conditions such as sickle cell disease, thalasse.

Guidelines for the Standard Monitoring of Patients With Thalassemia: Report of the Thal...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4511957
Journal of Pediatric Hematology/oncology; Tubman VN, Fung EB et. al.

Jul 22nd, 2015 - Chronic transfusion therapy has played a central role in extending life expectancy for patients with hemoglobinopathies such as thalassemia. However, this life-saving therapy is associated with numerous complications that now comprise the bulk of ...

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Drugs  69 results see all →

Clinicaltrials.gov  8,371 results

Surrogate Markers of Subclinical Atherosclerosis and Its Associated Factors in Patients...
https://doi.org/10.1080/03630269.2021.1907405
Hemoglobin Soltani D, Fakhrzadeh H et. al.

Apr 8th, 2021 - β-Thalassemia major (β-TM) is a severe genetic hemoglobin (Hb) disorder with cardiovascular complications such as atherosclerosis due to transfusion-dependent iron overload. We aimed to determine the associated factors with surrogate markers of su...

Hb Bart's Hydrops Fetalis Syndrome and Hb H Disease Caused by Deletional Chiang Rai (- ...
https://doi.org/10.1080/03630269.2021.1906269
Hemoglobin Ruengdit C, Khamphikham P et. al.

Apr 6th, 2021 - α0-Thalassemia (α0-thal) Chiang Rai (- -CR; NC_000016.10: g.144215_188843del) was identified as a novel 44.6 kb deletional type of α-thalassemia (α-thal), removing all α-like globin genes. However, little is known about the deleterious effects of ...

The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-th...
https://doi.org/10.1172/JCI144206
The Journal of Clinical Investigation; Matte A, Federti E et. al.

Apr 6th, 2021 - Anemia in β-thalassemia is related to ineffective erythropoiesis and reduced red cell survival. Excess free heme and accumulation of unpaired α-globin chains impose substantial oxidative stress on β-thalassemic erythroblasts and erythrocytes, impa...

see more →

News  179 results

FDA Places Clinical Hold on Sickle Cell Gene Therapy
https://www.medscape.com/viewarticle/946434

Feb 23rd, 2021 - Updated February 25, 2021 // Editor’s note: The original text has been corrected to accurately reflect the status of the patients with AML and MDS. The FDA placed a clinical hold yesterday on two gene therapy trials for sickle cell disease (SCD) a...

New Gene Therapy Vector Promising for Sickle Cell, Beta-Thalassemia
https://www.medscape.com/viewarticle/945626

Feb 10th, 2021 - NEW YORK (Reuters Health) - A new lentiviral gene therapy vector, ALS20, triggers more hemoglobin production at a lower dose than vectors currently used in trials of blood disorders such as sickle cell disease and beta-thalassemia, researchers say...

Fast Five Quiz: Are You Prepared to Confront Sickle Cell Disease?
https://reference.medscape.com/viewarticle/858964_2

Nov 1st, 2020 - Answer 1/5 Which of the following is the most common form of SCD? Your peers chose: HbS/b+ thalassemia 0% HbSC disease 0% HbS/HbE syndrome 0% HbS/SS disease 0% Figure 1. Sickle red blood cell. SCD refers to all genotypes containing at least one si...

Fast Five Quiz: Sickle Cell Disease Presentation and Diagnosis
https://reference.medscape.com/viewarticle/924856_4

Nov 1st, 2020 - It is very important to be able to distinguish sickle beta+ thalassemia from sickle trait. In the former, the amount of Hgb S is greater than the amount of Hgb A. In the latter, there is more Hgb A than Hgb S. People with sickle beta+ thalassemia ...

Clinical Practice Guidelines on CNS Complications in Sickle Cell Disease (ASH, 2020)
https://reference.medscape.com/viewarticle/931133

May 27th, 2020 - In April 2020, the American Society of Hematology published guidelines on the prevention, diagnosis, and treatment of central nervous system (CNS) complications in sickle cell disease.[1] Primary stroke prevention in children with sickle cell dise...

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Patient Education  15 results see all →