After a herculean data-gathering effort, in this issue of Blood, Aldenhoven and colleagues from Europe and North America provide an eye-opening assessment of long-term neurocognitive, organ, joint, and tissue function after allogeneic transplantation of children with mucopolysaccharidosis type I–Hurler syndrome (MPS-IH), along with an analysis defining a path to better these outcomes.
Keyphrases
- stem cell transplantation
- young adults
- bone marrow
- electronic health record
- replacement therapy
- big data
- bipolar disorder
- case report
- hematopoietic stem cell
- high dose
- cell therapy
- type diabetes
- stem cells
- data analysis
- low dose
- mesenchymal stem cells
- machine learning
- artificial intelligence
- weight loss
- smoking cessation