Whole-body metabolic modeling may be a promising approach to studying ultrarare mitochondrial disorders such as MNGIE or TK2d.

Mineralocorticoid receptor antagonists offer minimal incremental benefit in patients with ATTR-CM receiving disease-modifying therapy, a study shows.

None of the 9 IPF-specific quality-of-life instruments currently in use meet the criteria for a full Category A ...

Cord blood transplantation (CBT) conditioning intensity was associated with posttransplant outcomes that varied by age among ...

Experts in Germany, Austria, and Switzerland issued consensus recommendations for administering onasemnogene abeparvovec in ...

Ivosidenib monotherapy led to high response rates and prolonged survival in patients with IDH1-mutated myelodysplastic syndrome (MDS).

An AI model identified 5 categories of pathogenic processes that contribute to pulmonary involvement in neuronopathic Gaucher ...

Results of a real-world study found that icatibant is safe and effective for the treatment of acute HAE attacks.

In patients with infantile-onset Pompe disease, higher-dosage ERT regimens were linked to higher peaks in motor function, FVC, and strength.

Recent HR-MDS failures highlight the need to match trial populations, endpoints, and therapies to disease biology.

Concomitant IPF and lung cancer are associated with a substantially higher risk of mortality and respiratory health care burden, a study shows.

Telomere-related gene variants contribute to idiopathic pulmonary fibrosis (IPF) susceptibility, according to a new study.