Thalassaemia
A Kattamis, JL Kwiatkowski, Y Aydinok - The lancet, 2022 - thelancet.com
Thalassaemia is a diverse group of genetic disorders with a worldwide distribution affecting
globin chain synthesis. The pathogenesis of thalassaemia lies in the unbalanced globin …
globin chain synthesis. The pathogenesis of thalassaemia lies in the unbalanced globin …
Thalassaemia
AT Taher, DJ Weatherall, MD Cappellini - The Lancet, 2018 - thelancet.com
Inherited haemoglobin disorders, including thalassaemia and sickle-cell disease, are the
most common monogenic diseases worldwide. Several clinical forms of α-thalassaemia and …
most common monogenic diseases worldwide. Several clinical forms of α-thalassaemia and …
β-Thalassemias
AT Taher, KM Musallam… - New England Journal of …, 2021 - Mass Medical Soc
β-Thalassemias Defective synthesis of the β-globin chain causes recessively inherited
disorders characterized by inadequate hemoglobin production and chronic anemia …
disorders characterized by inadequate hemoglobin production and chronic anemia …
Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label …
KHM Kuo, DM Layton, A Lal, H Al-Samkari, J Bhatia… - The Lancet, 2022 - thelancet.com
Background Patients with non-transfusion-dependent thalassaemia (NTDT), although they
do not require regular blood transfusions for survival, can still accrue a heavy burden of …
do not require regular blood transfusions for survival, can still accrue a heavy burden of …
Clinical classification, screening and diagnosis for thalassemia
V Viprakasit, S Ekwattanakit - Hematology/Oncology Clinics, 2018 - hemonc.theclinics.com
Over the past decade, our knowledge of the clinical diagnosis and management of
thalassemia has progressed extensively. In recent years, the most critical change in clinical …
thalassemia has progressed extensively. In recent years, the most critical change in clinical …
Adrenal myelolipomas
J Calissendorff, CC Juhlin, A Sundin… - The lancet Diabetes & …, 2021 - thelancet.com
Adrenal myelolipomas are benign, lipomatous tumours with elements of myeloid cells, most
of which present as adrenal incidentalomas and comprise 3· 3–6· 5% of all adrenal masses …
of which present as adrenal incidentalomas and comprise 3· 3–6· 5% of all adrenal masses …
Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with β-thalassemia
A Piga, S Perrotta, MR Gamberini… - Blood, The Journal …, 2019 - ashpublications.org
Abstract β-thalassemia is a hereditary disorder with limited approved treatment options;
patients experience anemia and its complications, including iron overload. The study aim …
patients experience anemia and its complications, including iron overload. The study aim …
Beta thalassemia: new therapeutic options beyond transfusion and iron chelation
Hemoglobinopathies are among the most common monogenic diseases worldwide.
Approximately 1–5% of the global population are carriers for a genetic thalassemia …
Approximately 1–5% of the global population are carriers for a genetic thalassemia …
Thalassemias: an overview
M Angastiniotis, S Lobitz - International Journal of Neonatal Screening, 2019 - mdpi.com
Thalassemia syndromes are among the most serious and common genetic conditions. They
are indigenous in a wide but specific geographical area. However, through migration they …
are indigenous in a wide but specific geographical area. However, through migration they …
2021 update on clinical trials in β‐thalassemia
KM Musallam, R Bou‐Fakhredin… - American journal of …, 2021 - Wiley Online Library
The treatment landscape for patients with β‐thalassemia is witnessing a swift evolution, yet
several unmet needs continue to persist. Patients with transfusion‐dependent β …
several unmet needs continue to persist. Patients with transfusion‐dependent β …