Results 51 to 60 of about 9,026 (213)

Comparison of biochemical parameters of prevalent hemoglobinopathies with healthy individuals

open access: yesMedicine Science, 2020
Thalassemia is the most frequently seen monogenetic disorders around the world that is inherited as a recessive single-gene disease, resulting from mutations in α-or β-globin gene clusters.
Emrah Yerlikaya   +2 more
doaj   +1 more source

Investigation of Iron Deficiency, Iron Deficiency Anemia and Hemoglobinopathy Frequency in Kahramanmaraş City

open access: yes, 2022
The present study examined the levels of hemoglobin, MCV, ferritin, and hemoglobin electrophoresis of patients admitted to the hospital for different reasons, thus based on the results of these values, the number of patients diagnosed with iron ...
Duyuran, Rumeysa   +2 more
core   +1 more source

Splenic Tropism and Spleen‐Modulated Systemic Inflammation in Acute Plasmodium vivax Malaria

open access: yesAmerican Journal of Hematology, EarlyView.
ABSTRACT In chronic Plasmodium vivax (Pv) infection, the spleen accounts for over 98% of total‐body parasite biomass. Whether splenic tropism also occurs in acute infection and how the spleen influences pathogenesis have not been systematically explored. In Papua, Indonesia, we compared clinical and hematology data in 24 spleen‐intact and 25 previously
Steven Kho   +27 more
wiley   +1 more source

Health services use by children identified as heterozygous hemoglobinopathy mutation carriers via newborn screening [PDF]

open access: yes, 2021
Background Newborn screening (NBS) for sickle cell disease incidentally identifies heterozygous carriers of hemoglobinopathy mutations. In Ontario, Canada, these carrier results are not routinely disclosed, presenting an opportunity to investigate the ...
Robert J. Klaassen   +23 more
core   +1 more source

Precision medicine in paediatrics: Progress and priorities

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain   +3 more
wiley   +1 more source

A Nonhuman Primate Transplantation Model to Evaluate Hematopoietic Stem Cell Gene Editing Strategies for β-Hemoglobinopathies

open access: yesMolecular Therapy: Methods & Clinical Development, 2018
Reactivation of fetal hemoglobin (HbF) is a promising approach for the treatment of β-hemoglobinopathies and the targeting of genes involved in HbF regulation is under intensive investigation. Here, we established a nonhuman primate (NHP) transplantation
Olivier Humbert   +4 more
doaj   +1 more source

Evaluating the (comparative) safety profile of the novel oral polio vaccine type 2 using individual case safety reports in VigiBase

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Aim Novel oral polio vaccine type 2 (nOPV2) was used under the WHO emergency use listing for circulating vaccine‐derived polio virus (cVDPV) outbreaks from 2021 to 2023. We assessed nOPV2 adverse events following immunization (AEFIs) and compared its safety profile to other vaccines using VigiBase.
Comfort Kunak Ogar   +6 more
wiley   +1 more source

Cell and Gene Therapies Manufacturing Challenges and Integrated Good Manufacturing Practices Solutions: A Lifecycle Perspective

open access: yesBiotechnology and Bioengineering, EarlyView.
Lifecycle perspective on cell and gene therapy manufacturing challenges and enabling GMP solutions. ABSTRACT Cell and gene therapies (CGTs) are revolutionizing modern medicine; however, making these advanced medicines scalable and readily available to commercial manufacturers worldwide is a major challenge. The number of approved CGT products continues
Rajath Samaga   +2 more
wiley   +1 more source

Plummer–Vinson syndrome in a 10‐year‐old boy from Côte d'Ivoire: An exceptional paediatric case with African context

open access: yesJPGN Reports, EarlyView.
Abstract Plummer–Vinson syndrome (PVS) is characterised by the triad of dysphagia, iron‐deficiency anaemia, and proximal oesophageal webs. While well described in adults, paediatric cases remain exceptionally rare, particularly in sub‐Saharan Africa.
Paul Mike Tayou Mbobda   +2 more
wiley   +1 more source

Factors Contributing to Improved Bone Mineral Density in Patients With Transfusion‐Dependent Thalassemia

open access: yesThe Kaohsiung Journal of Medical Sciences, EarlyView.
ABSTRACT Advances in therapy have extended the life expectancy of patients with thalassemia to near that of the general population; complications such as endocrine disorders and osteoporosis remain prevalent. This study aimed to identify endocrine complications, factors associated with endocrine complications, and changes in bone mineral density (BMD ...
Cheng‐Ying Hsu   +7 more
wiley   +1 more source

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