Pancreatitis in patients with mitochondrial disease often presents in childhood, recurs, and occurs without conventional risk ...

Rates of meningococcal infection have stayed low and stable among US patients with MG or NMOSD treated with eculizumab or ...

No significant differences in the rates of autism or attention deficit hyperactivity disorder were observed between genetic ...

A mother whose son has TK2d says we need to keep collecting data as patients grow to reap the full potential of newly ...

An MCN of 54-67 and a UK-GR cytogenetic profile identified subgroups of children with high-hyperdiploid ALL and favorable outcomes.

The results of a retrospective study of patients with Gaucher disease (GD) type 1 may support earlier screening for Parkinson ...

Higher CD34+ cell and colony-forming unit-granulocyte/macrophage (CFU-GM) doses were associated with better DFS in high-risk ...

Age, comorbidity, disease risk, and donor type should guide conditioning intensity in myelodysplastic syndromes (MDS), rather ...

Researchers developed a single LRS assay that can detect and characterize genetic variations causing inherited myopathies, ...

The FDA has approved an update to the label of sotatercept-csrk to include data from a trial, which tested the drug in newly ...

RBCs in patients with low-risk MDS carry distinct physiological abnormalities that go beyond ineffective erythropoiesis.

Interstitial lung abnormalities have an adverse effect on long-term survival after non–small cell lung cancer resection, ...