Hemoglobinopathies are among the most common monogenic diseases worldwide.
Approximately 1–5% of the global population are carriers for a genetic thalassemia mutation. The …

… Musallam and Rayan Bou-Fakhredin contributed equally as first authors. … activator of the
RBC-specific form of PK (PK-R). Mitapivat has already shown efficacy and safety in clinical trials …

A significant amount of attention has recently been devoted to the mechanisms involved in
hemoglobin (Hb) switching, as it has previously been established that the induction of fetal …

Patients with non-transfusion-dependent thalassemia (NTDT) experience many clinical
complications despite their independence from frequent transfusions. Morbidities in NTDT stem …

To the Editor: The outbreak of Coronavirus Disease 2019 (COVID-19) caused by the novel
severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has led to a global health …

The pathogenesis of multiple sclerosis is characterized by a cascade of pathobiologic events,
ranging from focal lymphocytic infiltration and microglia activation to demyelination and …

Major advancements in transfusion programs and iron chelation therapy (ICT) administration
strategies have increased the life expectancy of b-thalassemia patients and improved their …

Full article: Improving outcomes and quality of life for patients with transfusion-dependent β-thalassemia:
recommendations for best clinical practice and the use of novel treatment …

Thalassemia incorporates a broad clinical spectrum characterized by decreased or absent
production of normal hemoglobin leading to decreased red blood cell survival and ineffective …

The COVID-19 pandemic has been deemed a global health emergency. Vaccination is
considered the most important and effective strategy to prevent infection and eradicate the …