Sickle cell anemia (SCA) was first described in the Western literature more than 100 years
ago. Elucidation of its molecular basis prompted numerous biochemical and genetic studies …

With advances in brain imaging and completion of randomized clinical trials (RCTs) for
primary and secondary stroke prevention, the natural history of central nervous system …

Background Sickle cell disease is characterized by the painful recurrence of vaso-occlusive
events. Gene therapy with the use of LentiGlobin for sickle cell disease (bb1111; …

The aim of this updated statement is to provide comprehensive and timely evidence-based
recommendations on the prevention of stroke among individuals who have not previously …

Silent cerebral infarct (SCI) is the most common form of neurologic disease in children with
sickle cell anemia (SCA). SCI is defined as abnormal magnetic resonance imaging (MRI) of …

Background: Sickle cell disease (SCD) is a life-limiting inherited hemoglobinopathy that
results in significant complications and affects quality of life. Hematopoietic stem cell …

Children with sickle cell disease experience organ damage, impaired quality of life, and
premature mortality. Allogeneic bone marrow transplant from an HLA-matched sibling can …

Curative therapy for individuals with severe sickle cell disease (SCD) who lack an HLA-
identical sibling donor has been frustratingly elusive. In with the goal of improving …

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

Abstract lovo‐cel (bb1111; LentiGlobin for sickle cell disease [SCD]) gene therapy (GT)
comprises autologous transplantation of hematopoietic stem and progenitor cells …