Drug Profiles discussed in this report include ACY-957, ALN-TMP, Antisense Oligonucleotides to Inhibit BCL11A and KLF1 for Hematological Disorders, BB-305, BtX-13, CNTO-530, Gene Therapy for Sickle Cell Disease and Beta Thalassemia
, Gene Therapy for Sickle Cell Disease and Thalassemia, glutamine, luspatercept, NiCord, PB-04, Peptide Agonists of Hepcidin for Beta-Thalassemia and Juvenile Hemochromatosis, Project x-1450, ruxolitinib phosphate, Small Molecule to Inhibit Histone Deacetylase for Beta-Thalassemi, sotatercept, Stem Cell Therapy for Beta Thalassemia
, Synthetic Peptides Hematological and Genetic Disorders, Thalagen.
occurs when similar gene defects affect production of the beta globin protein.
can cause shortness of breath, heart palpitations, fatigue, and enlarged bones due to the increased production of red blood cells in the bone marrow.
Frequency of Carriers for Beta Thalassemia
and Sickle Cell Trait in Mersin.
The traits, such as sickle trait (AS), beta thalassemia
trait, and alpha thalassemia trait, are particularly important in the prenatal patient, where it is vital to understand the likelihood of severe versus mild disease.
Tissue Doppler echocardiography reliably resects severity of iron overload in pediatric patients with beta thalassemia
Combined therapy with desferrioxamine and deferiprone in beta thalassemia
major patients with transfusional iron overload.
is a disease of the blood which can be treated only by regular blood transfusions which themselves will eventually kill the patient because of, among other things, a build-up of iron in the body.
Denaturing gradient gel electrophoresis and direct sequencing of PCR amplified genomic DNA: a rapid and reliable diagnostic approach to beta thalassemia
and translation of globin messenger RNA.
A 26-year-old female with history of sickle beta thalassemia
and smoking presented with one day of severe pleuritic chest pain and dyspnea following exercise.
Yersinia enterocolitica infection in a boy with beta thalassemia
The countries around the Mediterranean have many cases of beta thalassemia
Physicians use bone marrow transplants to treat a variety of blood diseases, from leukemia to potentially fatal genetic disorders such as beta thalassemia
The data presented at ASH demonstrate the potential for selective HDAC1/2 inhibition in hemoglobinopathies, including the two most prevalent severe human genetic diseases, sickle cell disease and beta thalassemia
, for which treatment options are severely limited," said Walter C.