Results 41 to 50 of about 102,550 (297)
Background Thalassemia is a common inherited hemoglobin disorder in Southeast Asia. Severe thalassemia can lead to significant morbidity for patients and economic strain for under-resourced health systems.
Julia Z. Xu+8 more
doaj +1 more source
Minihepcidins are rationally designed small peptides that mimic hepcidin activity in mice and may be useful for the treatment of iron overload [PDF]
Iron overload is the hallmark of hereditary hemochromatosis and a complication of iron-loading anemias such as β-thalassemia. Treatment can be burdensome and have significant side effects, and new therapeutic options are needed.
Alan Waring+16 more
core +2 more sources
Recent Cutting‐Edge Technologies for the Delivery of Peptide Nucleic Acid
This review provides an overview of PNA cellular delivery methods, starting with traditional peptide‐based systems and progressing to advanced approaches using nanoparticles, liposomes, and calixarene. It highlights how these innovative strategies have opened the way for more effective and efficient PNA delivery, ultimately enhancing the potential for ...
Concetta Avitabile+4 more
wiley +1 more source
Hematopoietic stem cell transplantation (HSCT) still remains the only definitive cure currently available for patients with thalassemia and sickle cell anemia.
Marco Marziali+3 more
doaj +1 more source
Third-degree heart block in thalassemia major: A case report [PDF]
Background: First and second-degree heart blocks are partly common rhythm disorders in thalassemic patients but complete heart block is a very rare complication of iron overload cardiomyopathy.
Hosseini, S.M., Maleki, A.R., Nikyar, B.
core +1 more source
Appraisal of patient-reported outcome measures in analogous diseases and recommendations for use in phase II and III clinical trials of pyruvate kinase deficiency [PDF]
Purpose: Pyruvate kinase deficiency (PKD) is a rare disease and understanding of its epidemiology and associated burden remains limited. With no current curative therapy, clinical manifestations can be life threatening, clinically managed by maintaining ...
Ionova, Tatyana+3 more
core +1 more source
Thirteen phosphorus balances were performed in four thalassemic children aged 6 to 10 years. No correlation was found between phosphorus intake and serum level or between phosphorus intake and net absorption. There was a positive correlation among daily phosphorus intake, net absorption, and 24-hour urinary excretion.
Lapatsanis, P.+4 more
openaire +4 more sources
Dysregulated proteolysis mediated by kallikrein‐related peptidases (KLKs) and iron overload are involved in the progression of neurodegenerative diseases. Deferasirox, an clinically‐approved iron chelator, and its newly synthesized derivatives have been identified as inhibitors of major central nervous system KLKs with low cytotoxicity and effective ...
Rilès Boumali+11 more
wiley +1 more source
Background Thalassemia, an inherited hemoglobin disorder, has become a global public health problem due to population migration. Evidence-based strategies for thalassemia prevention in migrants are lacking.
Julia Z. Xu+6 more
doaj +1 more source
Identification of erythroferrone as an erythroid regulator of iron metabolism. [PDF]
Recovery from blood loss requires a greatly enhanced supply of iron to support expanded erythropoiesis. After hemorrhage, suppression of the iron-regulatory hormone hepcidin allows increased iron absorption and mobilization from stores.
Ganz, Tomas+5 more
core +1 more source