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 ...
An AI model identified 5 categories of pathogenic processes that contribute to pulmonary involvement in neuronopathic Gaucher ...
Experts in Germany, Austria, and Switzerland issued consensus recommendations for administering onasemnogene abeparvovec in ...
Whole-body metabolic modeling may be a promising approach to studying ultrarare mitochondrial disorders such as MNGIE or TK2d.
Results of a real-world study found that icatibant is safe and effective for the treatment of acute HAE attacks.
Ivosidenib monotherapy led to high response rates and prolonged survival in patients with IDH1-mutated myelodysplastic syndrome (MDS).
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.
Mineralocorticoid receptor antagonists offer minimal incremental benefit in patients with ATTR-CM receiving disease-modifying therapy, a study shows.
After applying for accelerated FDA approval of delpacibart zotadirsen in DMD, Avidity launched a confirmatory trial of the ...
Telomere-related gene variants contribute to idiopathic pulmonary fibrosis (IPF) susceptibility, according to a new study.
Some results have been hidden because they may be inaccessible to you
Show inaccessible results