Rund D, Rachmilewitz E. New trends in the treatment of beta-thalassemia. Crit Rev Oncol Hematol 2000;33(2):105-18.Rund D, Rachmilewitz E. New trends in the treatment of beta-thalassemia. Crit Rev Oncol Hematol 2000; 33: 105-118....
Two drugs gained approval for the treatment of Duchenne muscular dystrophy (DMD), a rare X-linked genetic neuromuscular disorder resulting from mutations in the DMD gene, which encodes dystrophin, a protein crucial for proper muscle function (Keam2023f). One of these drugs isvamorolone, a synth...
The structure of EDTA has been modified by replacing two acetate groups with o-hydroxybenzyl groups, producing N,N′-bis- o-hydroxybenzylethylenediamine-N,N′-diacetric acid (HBED) [1] which has a very high affinity for the Fe(III) ion. Previously, an analogous ligand, ethylenebis- o-hy...
In Vivo Developing Immunotherapy of Ex Vivo Expansion of Human Natural Killer Cells from Thalassemia Patients for Leukaemia Author(s): M. Alkhaled*, A. M. Elazzazy, O. A. Almaghrabia and R. Handgretinger DOI: 10.36468/pharmaceutical-sciences.spl.451 Abstract Full-Text PDF Research...
Beta thalassemia and hemoglobin (HbE)-related hemoglobinopathies are common public health problems in developing countries. High-performance liquid chromat... A Nandi,M Talukdar,S Bhattacharya,... - 《Indian Journal of Pathology & Microbiology》 被引量: 0发表: 2024年 Critical success factors for ...
a treatment for sickle cell disease and beta thalassemia, two blood disorders. It was developed by CRISPR Therapeutics, the Swiss company co-founded by Nobel laureate Emmanuelle Charpentier, and Vertex Pharmaceuticals, a large Boston-based biotech firm. The treatment wasapproved by U.K. regulatorsth...
Invasive Yersiniosis in a Pediatric Patient With beta-Thalassemia Major: Acute Decompensation After Rapid Blood Transfusion. Pediatr Emerg Care. 2019;35(7):e118–20. https://doi.org/10.1097/PEC.0000000000001873. Article PubMed Google Scholar ECDC. Annual epidemiological report for 2022. Stockholm:...
Finally, although the search for the ideal vector continues, only the outcomes of the many preclinical and clinical studies will determine what this vector will look like in the near future. Show abstract Combining gene therapy and fetal hemoglobin induction for treatment of β-thalassemia 2013, ...
The first two patients with beta thalassaemia no longer need blood transfusions since being treated 15 and five months ago. Nor does the patient with sickle cell disease, nine months after treatment. The results are excellent, says Marina Cavazzana at the Necker-Enfants Malades Hospital ...
The expenses involved in making sure a child with thalassemia gets the best medical care possible over the course of that child’s lifetime can be enormous. There may be a significant difference in the treatment and services that private medical insurance, a public school system, or such progra...