Thalassemia was first clinically described nearly a century ago and treatment of this
widespread genetic disease has greatly advanced during this period. DNA‐based diagnosis …

Diamond‐Blackfan anaemia (DBA) is a rare inherited marrow failure disorder, characterized
by hypoplastic anaemia, congenital anomalies and a predisposition to cancer as a result of …

Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment
for some inherited disorders, including selected primary immunodeficiencies (PIDs). In the …

Krabbe disease is a rare neurodegenerative disorder caused by a deficiency in
galactocerebrosidase. The only effective treatment is hematopoietic stem cell transplantation …

Hematopoietic stem cell transplantation from HLA-matched sibling donors results in disease-
free survival of> 90% in patients with sickle cell disease (SCD); however, only approximately …

Allogeneic hematopoietic stem cell transplantation (HSCT) is curative for many
nonmalignant pediatric disorders, including hemoglobinopathies, bone marrow failure …

Alemtuzumab, fludarabine, and melphalan reduced-intensity conditioning (RIC) regimens
are increasingly used for the hematopoietic cell transplantation (HCT) of pediatric and young …

The success of transplant procedures in patients with beta-thalassemia major (β-
thalassemia) goes hand-in-hand with improvements in disease knowledge, better …

Following the advent of molecular assays that measure T cell receptor excision circles
(TRECs) present in recent thymic emigrants, it has been conclusively shown that …

Umbilical cord blood (UCB) is now widely used as an alternative hematopoietic stem cell
source for patients lacking closely matched related or unrelated adult donors. UCB …