Results 131 to 140 of about 2,697,911 (252)

Phase 3, randomised, placebo‐controlled trial of voxelotor for the resolution of leg ulcers in sickle cell disease: RESOLVE

British Journal of Haematology, EarlyView.
In this trial, conducted in Kenya, Nigeria and Brazil, the resolution of leg ulcers was similar between the voxelotor and placebo arms. In the 12‐week blinded period, the number of sickle cell anaemia with crisis events was higher in the voxelotor arm compared to placebo.
Bernhards Ogutu, Janet Oyieko, Halima Bello‐Manga, Adeseye Akinsete, Jessie Githanga, José Valber Lima Meneses, Carolyn Hoppe, Mark W. Davis, Jeremy Rupon, Ching‐Ray Yu, Roberto Pinto, Elena Tudone, Ibrahim Musa Idris, Martin Meremikwu, Aderson da Silva Araújo, Rodolfo D. Cançado, Guilherme Fonseca, Patrícia Santos Resende Cardoso, Obiageli Eunice Nnodu, on behalf of the RESOLVE Investigators   +19 more
wiley   +1 more source

The β-thalassemia carrier status of fetal biological parents.

, 2023
The β-thalassemia carrier status of fetal biological parents.
Xiaojing Wu (337665), Yayun Ling (15183299), Ping Chen (89712), Hengying Zhu (15183296), Wenguang Jia (15183290), Jiying Shi (15183293)   +5 more
core   +1 more source

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

Nature Medicine, 2020
β-Thalassemia pathology is due not only to loss of β-globin (HBB), but also to erythrotoxic accumulation and aggregation of the β-globin-binding partner, α-globin (HBA1/2).
M. Cromer, J. Camarena, Renata M. Martin, B. Lesch, Christopher A. Vakulskas, Nicole M. Bode, G. Kurgan, Michael A. Collingwood, G. Rettig, M. Behlke, V. Lemgart, Yankai Zhang, Ankush Goyal, Feifei Zhao, Ezequiel Ponce, W. Srifa, R. Bak, N. Uchida, R. Majeti, V. Sheehan, J. Tisdale, D. Dever, M. Porteus   +22 more
semanticscholar   +1 more source

Optimising Haematopoietic Stem Cell Transplantation: Enhancing Myeloablation Sensitivity and Alleviating Anaemia Using Roxadustat (FG‐4592)

Cell Proliferation, EarlyView.
The use of FG‐4592 in the process of myeloablation can promote haematopoietic cell apoptosis for more effective myeloablation, maintain the number of RBC and may create a pluripotent microenvironment suitable for donor cells, so as to enable more effective haematopoietic reconstruction of donor cells in vivo.
Guanheng Yang, Jiahui Wu, Xinbing Guo, Wenchen Shen, Xiao Sang, Xiuli Gong, Shu‐Zhen Huang, Jingzhi Zhang, Fanyi Zeng   +8 more
wiley   +1 more source

A Population-Oriented Genetic Scoring System to Predict Phenotype: A Pathway to Personalized Medicine in Iraqis With β-Thalassemia

To assess the roles of genetic modifiers in Iraqi β-thalassemia patients, and determine whether a genotype-based scoring system could be used to predict phenotype, a total of 224 Iraqi patients with molecularly characterized homozygous or compound ...
Regir K Sadullah (18015372), Adil Abozaid Eissa (18015375), Sulav D Atroshi (18015369), Shatha Qadir (18015378), Shaima Al-Zebari (16730631), Dilan Khalil (18015381), Nasir Al-Allawi (3095073), Gernot Kriegshäuser (5680346), Christian Oberkanins (18015384)   +8 more
core   +1 more source

Vitamin and mineral supplementation for β-thalassemia during COVID-19 pandemic

, 2020
Aim: Low levels of immune-related micronutrients have been identified in β-thalassemia samples. Moreover, the excess amount of iron, contributing to oxidative stress in the pathogenesis of the disease, alters the immune system in β-thalassemia, which is ...
Yovani Savitri, Annette d'Arqom, Melvanda G Putri, Andi Muh Rahul Alfaidin   +3 more
core   +1 more source

Metabolomic Investigation of β-Thalassemia in Chorionic Villi Samples [PDF]

, 2019
Beta-thalassemias are blood disorders characterized by poorly understood clinical phenotypes ranging from asymptomatic to severe anemia. Metabolic composition of the human placenta could be affected by the presence of pathological states such as β ...
Valentina Corda, Antonella Balsamo, Iuculano, Ambra, Corda, Valentina, Luigi Atzori, Laura Tronci, Atzori, Luigi, Giovanni Monni, Monni, Giovanni, Tronci, Laura, Federica Murgia, Balsamo, Antonella, Ambra Iuculano, Murgia, Federica, Peddes, Cristina, Cristina Peddes   +15 more
core   +1 more source

Cascade Screening of β-Thalassemia in an Indian Family Using Flow Injection Analysis–Triple Quadrupole Mass Spectrometry: Comparison of Micro Sampling Approaches with Conventional Electrophoresis

Thalassemia Reports
Background: β-thalassemia is a rare genetic disorder affecting 1–5% of the global population and poses a health burden due to migration of individuals from endemic regions. Identifying asymptomatic β-thalassemia carriers is essential to prevent the birth
Ankitha K. Puthiyaveettil, Harshini K. Musuvathi, Deepalakshmi D. Putchen   +2 more
doaj   +1 more source

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source

Alternative options for DNA-based experimental therapy of β-thalassemia.

, 2012
INTRODUCTION: Beta-thalassemias are caused by more than 200 mutations of the β-globin gene, leading to low or absent production of adult hemoglobin. Achievements have been made with innovative therapeutic strategies for β-thalassemias, based on research ...
GAMBARI, Roberto
core   +1 more source