Researchers advanced the therapy with an AI platform that uses machine learning models trained on patient endotyping, multiomics, biomarker, and clinical data. The US Food and Drug Administration (FDA ...

In a pooled analysis of 48 emulated trials, the mortality risk was 69.7% lower among individuals who received an exon-skipping therapy plus glucocorticoids than among those who received ...

The mixed vascular-fibrotic group demonstrated the shortest median progression-free survival (36 months) and the highest independent risk of disease progression. Researchers proposed a new ...

Arm span gains of 9.4 cm in treatment-naive and 7.9 cm in navepegritide-experienced children over 52 weeks surpassed the natural history trend for untreated children with achondroplasia. Children with ...

All 24 participants in the phase 1b trial maintained their baseline motor milestones, and half achieved at least 1 new WHO motor milestone after salanersen treatment. The US Food and Drug ...

The majority (74%) of patients with PBC treated with seladelpar achieved composite alkaline phosphatase normalization at 24 months. Seladelpar treatment is associated with high and sustained rates of ...

A panel of 88 specialists reached a consensus that primary biliary cholangitis-autoimmune hepatitis should be defined as a variant and not an overlap syndrome. A new Delphi initiative has established ...