Results 141 to 150 of about 2,702 (213)

Long‐Term Endocrine Effects of Hematopoietic Stem Cell Transplantation in Children: A Reappraisal

Clinical Transplantation, Volume 40, Issue 3, March 2026.
ABSTRACT Background Hematopoietic stem cell transplantation (HSCT) is widely used in both malignant and non‐malignant diseases in children. This study aimed to evaluate long‐term endocrine complications in pediatric HSCT survivors. Methods Children who underwent HSCT between April 2010 and October 2014 were retrospectively assessed.
Dilek Gürlek Gökçebay, Gamze Gürsoy, Mehtap Kanbur, İkbal O. K. Bozkaya, Ozlem Arman Bilir, Namik Yasar Ozbek   +5 more
wiley   +1 more source

Severe Pain Phenotype in Hemoglobin C (HbC)/Beta-Zero (β⁰) Thalassemia Without Hemoglobin S (HbS): A Clinical and Diagnostic Challenge. [PDF]

Cureus
Martinez MJ, Friedland BN, Jha A, Zagoruychenko T, Berman S.   +4 more
europepmc   +1 more source

Making Advanced Therapies Affordable and Accessible: Two Strategic Approaches

Developing World Bioethics, Volume 26, Issue 1, Page 66-78, March 2026.
ABSTRACT This article explores two complementary strategies for addressing the affordability and access challenges facing advanced therapies. As high development costs and limited market access have led to the withdrawal of several therapies, the article examines how these barriers create ‘valleys of death’ that prevent innovation from reaching ...
Ubaka Ogbogu, Lauren Albrecht
wiley   +1 more source

Structural and clinical characterization of Hb Móstoles (HBA2:c.176A>G; p.His59Arg): a new unstable alpha-globin variant with thalassemic features. [PDF]

Am J Blood Res
Ferrer-Benito S, Ordoñez M, Bermejo A, Martínez JM, Ortega B, González FA, Villegas A, Benavente C, Ropero P.   +8 more
europepmc   +1 more source

Pubertal Assessment and Growth in Patients With Hemoglobinopathies: A Longitudinal Multicenter Study on the Association With Ferritin Levels

European Journal of Haematology, Volume 116, Issue 3, Page 276-289, March 2026.
ABSTRACT Objectives Although Advancements in the Treatment of Hemoglobinopathies have Considerably Increased Life Expectancy, Hormonal and Pubertal Development Have Been Continuously Affected by Complications From Transfusion‐Related Iron Overload and Cytotoxic Therapies.
J. Dülberg, C. Sanchez, M.‐A. Burckhardt, L. Alacán Friedrich, V. Salow, A. Radauer‐Plank, A. Borgmann‐Staudt, H. Cario, M. Diepold, B. Drexler, L. Infanti, S. Kroiss, N. Dietliker, R. Merki, L. Njue, L. Oevermann, A. Rovó, K. Scheinemann, M. Schneider, M. Balcerek, T. Diesch‐Furlanetto   +20 more
wiley   +1 more source

Evaluation of β-thalassemias in the premarital hemoglobinopathy screening program: A retrospective study. [PDF]

Pak J Med Sci
Goktas O.
europepmc   +1 more source

Cas9 beyond CRISPR – SUMOylation, effector‐like potential and pathogenic adaptation

The FEBS Journal, Volume 293, Issue 5, Page 1285-1296, March 2026.
This Viewpoint explores the possibility that Cas9, beyond its canonical role in CRISPR immunity, may act as a host‐modulating effector during infection by Cas9‐encoding bacteria. Such a nucleomodulin‐like function could be influenced by eukaryotic post‐translational modifications, particularly SUMOylation, with potential implications for host–pathogen ...
Umut Sahin
wiley   +1 more source

Relevance of Next-Generation Sequencing in the Diagnosis of Thalassemia and Hemoglobinopathies: The Experience of Four Italian Diagnostic Hubs. [PDF]

Genes (Basel)
Selvatici R, Guida V, Maffei M, Irrera MA, Margutti A, Bisceglia P, Mogni M, Melchionda E, Stoico G, Grifone N, Bocciardo L, Salerio S, Nagliati V, Alberico A, Tringali G, Melles C, De Luca A, Ferlini A, Coviello D, Curcio C.   +19 more
europepmc   +1 more source

Cell and Gene Therapy in Equine Ocular Disease

Veterinary Ophthalmology, Volume 29, Issue 2, March 2026.
ABSTRACT Equine ocular disease is common and often challenging to treat using traditional methods. This has led to the development of new therapies. Like human medicine, veterinary medicine is adopting cellular and gene therapy as innovative approaches. Equine ocular disease is a particularly promising area for these techniques.
Kimberly A. S. Young, Lauren V. Schnabel, Brian C. Gilger   +2 more
wiley   +1 more source

Epidemiological Profile of Haemoglobinopathies in Different Districts of West Bengal: A Retrospective Study. [PDF]

Niger Med J
Dhar O, Sharma A, Bhaumik S, Das AK.
europepmc   +1 more source