Results 121 to 130 of about 4,160 (239)

NOVEL THERAPEUTIC APPROACHES IN THALASSEMIAS, SICKLE CELL DISEASE AND OTHER RED CELL DISORDERS

A deeper understanding of the pathophysiology of hereditary red cell disorders and the development of novel classes of pharmacologic agents in this last decade have provided novel therapeutic approaches to thalassemias, sickle cell disease (SCD) and ...
Lucia De Franceschi, Filippo Mazzi, Valeria Maria Pinto   +2 more
core  

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

British Journal of Haematology, Volume 209, Issue 5, Page 2252-2263, November 2026.
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

β-Thalassemias

New England Journal of Medicine, 2021
Ali T, Taher, Khaled M, Musallam, M Domenica, Cappellini   +2 more
openaire   +6 more sources

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

Cell Proliferation, Volume 59, Issue 11, November 2026.
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

Smartphone‐Based Teledentistry to Support Clinical Triage and Risk‐Informed Dental Care in Patients With Inherited Bleeding and Haemoglobin Disorders: A Cross‐Sectional Diagnostic Agreement Study

Haemophilia, Volume 32, Issue 6, Page 1378-1386, November/December 2026.
ABSTRACT Introduction Patients with inherited bleeding and haemoglobin disorders face barriers to accessing timely dental care, increasing the risk of untreated oral disease and complications related to invasive procedures. Aim To evaluate the agreement between smartphone‐based asynchronous teledentistry and face‐to‐face examination for oral conditions,
Victor Cordeiro da Silva, Carolina Mendes Frusca do Monte, Leandro Tavares da Silva, Gabriela Silva Almeida, Claudia Santos Lorenzato, José Vitor Nogara Borges de Menezes, Fabian Calixto Fraiz, Cassius Carvalho Torres‐Pereira   +7 more
wiley   +1 more source

Thalassemias and Other Hemoglobinopathies in Former Yugoslavia

Balkan Journal of Medical Genetics, 2008
Efremov G
doaj   +1 more source

15 years of hemoglobinopathies and thalassemias: from a textbook example in biochemistry to an everyday diagnostic challenge

Journal of Laboratory Medicine
Hemoglobinopathies are among the most common monogenic disorders worldwide and are a significant global health problem. However, diagnosing these disorders, especially those involving complex genotypes (HBA/HBB), is challenging.
Häuser Friederike, Beck Joachim, Mildenberger Eva, Faber Jörg, Skevaki Chrysanthi, Beck Olaf, Rossmann Heidi   +6 more
doaj   +1 more source

Clinical Spectrum, Outcomes, and Factors Associated With Encephalopathy in Children With Influenza‐Associated Neurological Complications: A Retrospective Cohort Study

Influenza and Other Respiratory Viruses, Volume 20, Issue 11, November 2026.
ABSTRACT Objective This study aims to characterize the clinical spectrum, outcomes, and factors associated with influenza‐associated encephalopathy in hospitalized children with influenza‐associated neurological complications. Methods This retrospective cohort study included children younger than 18 years who were hospitalized with laboratory‐confirmed
Süleyman Şahin, Gonca Başak Soran, Büşra Kaygusuz Aydemir, Fatma Sargin, Mustafa Gençeli, Abdullah Akkuş, Halil Çelik, Özge Metin Akcan, Ahmet Sami Güven   +8 more
wiley   +1 more source

Molecular aspects of alpha dn beta thalassemias [Aspectos moleculares de las talasemias alpha y beta.]

, 1990
Thalassemia has been considered a recessive, autosomic, hereditary disease, characterized by microcytic, hypochromic, hemolytic anemia, which occurs as the consequence of a defect in the synthesis of the globin chains, the two most frequent types are ...
Ibarra, B., Hernandez-Cordova, A., Perea, F.J.   +2 more
core   +1 more source

Hemoglobinopathies types and their peculiarities in India

Hemoglobinopathies, including thalassemias and sickle cell disorders are among the most prevalent inherited autosomal recessive blood disorders globally and pose significant public health challenges in India.
Ткаченко, Олена Вікторівна, Tkachenko, О. V., Sharma, D.   +2 more
core