Results 111 to 120 of about 140,652 (287)

HOPE Kids 2: Phase 3, Randomized Trial of Voxelotor in Children With SCD and Conditional Cerebral Blood Flow Velocities

American Journal of Hematology, Volume 101, Issue 11, Page 2799-2811, November 2026.
ABSTRACT Children with SCD have elevated stroke risk, correlated with cerebral blood flow velocity (CBFV). HOPE Kids 2 was a phase 3, multicenter, double‐blind, placebo‐controlled trial evaluating the effect of voxelotor on CBFV. Participants aged 2 to < 15 years with SCD (HbSS/HbSβ0) and conditional CBFV (170 to < 200 cm/s) were randomized 1:1 to ...
Halima Bello‐Manga, Shehu Umar Abdullahi, Hoda Hassab, Biobele Brown, Videlis Nduba, Jessie Githanga, Adeseye Michael Akinsete, Amal El‐Beshlawy, Diana Hanna, Catherine Segbefia, Iheanyi Okpala, Azza Tantawy, Bernhards Ogutu, Venée N. Tubman, Vivian Paintsil, Yasser Wali, Mohammed Essa, Paul Telfer, Sandy Dixon, Anne Hickman, Jennifer F. Doss, Mark W. Davis, Jeremy Rupon, Fenella J. Kirkham, R. Clark Brown, on behalf of the HOPE Kids 2 Investigators, Amal El‐Beshlawy, Mohamed Badr, Diana Hanna*, Hoda Hassab, Azza Tantawy, Vivian Paintsil, Catherine Segbefia, Raffaella Colombatti, Daniela Cuzzubbo, Silverio Perrotta, Jessie Githanga, Videlis Nduba, Bernhards Ogutu, Shehu Umar Abdullahi, Adeseye Michael Akinsete, Halima Bello‐Manga, Biobele Brown, Iheanyi Okpala, Yasser Wali, Mohammed Essa, Banu Kaya, Paul Telfer, Ofelia Alvarez, Natasha Archer, Shayla Bergmann, R Clark Brown, Andrew Campbell, Carlton Dampier*, Richard Hurwitz, Erin Jackson, Janet Kwiatkowski, Melissa M. McNaull*, Venée N. Tubman   +58 more
wiley   +1 more source

Clinical and Laboratory Study of Sickle Cell/β-Thalassemia [PDF]

, 1986
We have studied 39 patients doubly heterozygous for sickle cell/β-thalassemia, 12 with sickle cell/βo- thalassemia and 27 with sickle cell/β+-thalassemia. Generally, sickle cell/βo-thalassemia is considered more severe than sickle cell/β+-thalassemia. In
Maeda, Koichi, Van Slyck, Ellis J., Hawley, Robert C.   +2 more
core   +1 more source

The emerging role of non-coding RNAs in the pathogenesis of infantile hemangioma

Cancer Cell International
Infantile hemangioma (IH) is a common benign tumor that occurs in children, affecting both blood vessels and soft tissues. Its pathological features include abnormal proliferation of endothelial cells and an irregular vascular structure.
Najmaldin Saki, Narjes Sadat Sadati, Zeinab Deris Zayeri, Masoud Kargar   +3 more
doaj   +1 more source

Expanding the Utility of Exome Sequencing in Preventive and Population Genetics

American Journal of Medical Genetics Part A, Volume 200, Issue 11, Page 2433-2442, November 2026.
ABSTRACT Carrier screening is a long‐standing genetic testing process offered to at‐risk couples, with or without a family history, who might have pregnancies affected by an autosomal recessive (AR) or X‐linked (XL) disorder. A total of 276 unrelated individuals, initially referred for rare disorder screening by clinicians, were enrolled in this study ...
Charilaos Kostoulas, Athanasia Sesse, Ioanna Bouba, Robert Najdecki, Spyridon Konitsiotis, Sofia Markoula, Ioannis Georgiou   +6 more
wiley   +1 more source

Intratumoral and Peritumoral Functional Connectivity Reveals Network‐Level Signatures of Glioma

MedComm, Volume 7, Issue 11, November 2026.
Using resting‐state fMRI in 306 glioma patients, this study maps individualized intra‐ and peritumoral functional networks. Novel metrics (RIFR, RPTR) quantify tumor‐related network remodeling, correlating with pathology, structure, cognition, and survival. A prognostic model incorporating these network features achieves an AUC of 0.90.
Hongbo Bao, Yan Bai, Jie Hu, Wencai Ding, Jiacheng Lai, Andrew Zalesky, Ye Ella Tian, Yanmei Tie, Janet Mei, Shengyu Fang, Xing Fan, Licia P. Luna, Craig K. Jones, Jianxun Ren, Weigang Cui, Jiadong Yan, Zeya Yan, Zihan Wang, Renwu Zhang, Zhonghua Lv, Yihang Jiao, Tao Jiang, Peng Liang, Haris I. Sair, Yinyan Wang   +24 more
wiley   +1 more source

Enhanced induction of fetal hemoglobin by the combination of decitabine with RN-1 in β-thalassemia/HbE erythroid progenitor cells

Molecular Medicine
Background Fetal hemoglobin (HbF; α2γ2) induction is a well-established approach for β-hemoglobinopathies, including sickle cell disease (SCD) and β-thalassemia.
Tiwaporn Nualkaew, Phitchapa Pongpaksupasin, Thongperm Munkongdee, Nattrika Buasuwan, Kittiphong Paiboonsukwong, Orapan Sripichai, James Douglas Engel, Suradej Hongeng, Suthat Fucharoen, Natee Jearawiriyapaisarn   +9 more
doaj   +1 more source

Anemia in a Cohort of Greek Patients with Sickle Cell Disease: Clinical Insights and Therapeutic Challenges

Hematology Reports
Background/Objectives: Sickle cell disease (SCD) is a multi-systemic hemoglobinopathy affecting many patients worldwide. Clinical manifestations include vaso-occlusive (VOC), aplastic, or hemolytic crises. Anemia of a complex etiology is also very common.
Michael D. Diamantidis, Ioanna Argyrakouli, Efthymia Betsi, Alexandra Roupakia, Petroula Krestinidi, Maria Palassopoulou, Omar Zayed, Lazaros Karaloizos, Achilles Manafas   +8 more
doaj   +1 more source

Prevalence and Distribution of Thalassemia Trait Screening

, 2017
Thalassemia is an inherited disorder of autosomal recessive gene caused by decrease or absent production of one or two type of globin chain. This disorder will affect the quality and quantity of blood production.
Leonard, Elory, Handayani, Niken Satuti Nur, Husna, Nailil, Sanka, Imanuel, Putri, Chintya, Al Arif, Ahmad   +5 more
core   +1 more source

The role of iron in normal and impaired testicular function

Andrology, Volume 14, Issue 8, Page 2373-2387, November 2026.
Abstract Iron plays a critical role in testicular physiology, impacting spermatogenesis, testosterone production, and overall testicular function. Iron homeostasis is maintained through systemic and cellular regulatory mechanisms, including hepcidin‐mediated systemic iron control and the iron‐responsive element/iron regulatory protein (IRE/IRP) system ...
Aileen Harrer, Esther G. Meyron‐Holtz, Andreas Meinhardt   +2 more
wiley   +1 more source

Loss of alpha-hemoglobin-stabilizing protein impairs erythropoiesis and exacerbates beta-thalassemia

, 2004
Hemoglobin (Hb) A production during red blood cell development is coordinated to minimize the deleterious effects of free α- and β-Hb subunits, which are unstable and cytotoxic.
Weiss, MJ (15531407), Foster-Brown, L (15531449), Kihm, AJ (15531443), David Gell (14737321), Katein, AM (15531422), Adachi, K (15531446), Mackay, JP (15531410), Louden, CS (15531452), Zhou, S (15524792), Yu, X (5802800), Kong, Y (15531413), Gow, AJ (15483407)   +11 more
core