Nasdaq: SGMO), the leader in therapeutic genome editing, announced the presentation of data from its ZFP Therapeutic programs, including data demonstrating the production of therapeutic levels of Factor IX (FIX) clotting protein in non-human primates (NHPs) for the treatment of hemophilia B and disruption of the BCL11A Enhancer for the treatment of hemoglobinopathies
Genetic disorders, and in particular hemoglobinopathies
such as sickle cell anemia and thalassemia are common in Saudi Arabia, especially in the eastern and southern regions.
The G8 utilizes gold standard ion-exchange HPLC with visual identification of hemoglobinopathies
Furthermore, the bill seeks authorization for the Centers for Disease Control (CDC) to be the funding agency for the continuance and establishment of the Hemoglobinopathies
Surveillance System program and SCD public health promotion initiatives.
Twenty-seven additional chapters treat erythropoiesis, thalassemia, sickle cell anemia, and hemoglobinopathies
, their world distribution and health burden.
Beyond transplant, Regenerex's novel platform potentially has curative potential for multiple underserved diseases and will be investigated in the rescue of serious genetic deficiencies such as inherited metabolic storage disorders and hemoglobinopathies
The detecting methods for HbAlc have the potential of detecting coexisting Hb variants or hemoglobinopathies
The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-AT3 for the treatment of hemophilia and rare bleeding disorders (RBD), ALN-AS1 for the treatment of acute intermittent porphyria, ALN-PCS for the treatment of hypercholesterolemia, and ALN-TMP for the treatment of hemoglobinopathies
are amongst the most prevalent of all human genetic disorders resulting from structural changes or abnormal synthesis of polypeptide chain of the hemoglobin molecule (1).
were confirmed by electrophoresis (Paragon Hemoglobin electrophoresis, Acid Hemoglobin electrophoresis, Beckman Coulter, Fullerton, CA).
Guideline: the laboratory diagnosis of hemoglobinopathies
imply structural abnormalities in the globin proteing themselves.