Results 151 to 160 of about 219,391 (317)
British Journal of Haematology, EarlyView.In this post hoc analysis of the phase 2 BEYOND trial, the majority of patients with non‐transfusion‐dependent β‐thalassaemia achieved clinically meaningful haemoglobin levels ≥10.0 g/dL and increases from baseline ≥1.0 g/dL, thresholds associated with reduced risk of morbidity and mortality and recommended as an indication and target for treatment by ...
Khaled M. Musallam +11 more
wiley +1 more source
Early Kidney Damage Markers after Deferasirox Treatment in Patients with Thalassemia Major: A Case-Control Study [PDF]
, 2019 Hamidreza Badeli +9 more
openalex +1 more source
Making Advanced Therapies Affordable and Accessible: Two Strategic Approaches
Developing World Bioethics, EarlyView.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
Health Care Challenges of Hereditary Common Hematological Disorders in Odisha, India [PDF]
, 2012 Medical Genetics over the past few decades have emerged as an important and powerful medical specialty with increasing appreciation of its role and function in the biomedical sciences.
Balgir, RS
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European Journal of Haematology, EarlyView.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 +20 more
wiley +1 more source
Intervention and Prevention of Hereditary Hemolytic Disorders in Two Ethnic Communities of Sundargarh District of Orissa, India: An Experience from KAP Studies [PDF]
, 2010 Hereditary hemolytic disorders are important public health challenges in India. They cause a high degree of morbidity, mortality and fetal wastage in vulnerable communities.
Balgir, RS
core +1 more source
Cas9 beyond CRISPR – SUMOylation, effector‐like potential and pathogenic adaptation
The FEBS Journal, EarlyView.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
Case report: Rethinking NGS analysis in diagnosing Diamond-Blackfan anemia syndrome
Frontiers in GeneticsDiamond-Blackfan anemia syndrome (DBAS) is a rare inherited bone marrow failure (BMF) syndrome characterized by erythroid aplasia, congenital malformations, and cancer predisposition.
Panayiota L. Papasavva +7 more
doaj +1 more source