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 ...

No ICANS or deaths occurred, and all serious adverse events resolved without sequelae. Cizutamig, a T-cell engager, is generally safe and well tolerated in generalized myasthenia gravis (MG) and other ...

Z-rostudirsen is designed to enable production of near full-length dystrophin in both muscle and the central nervous system, unlike similar therapies that target muscle alone. Dyne Therapeutics has ...

In 74 patients with IPF receiving antifibrotic therapy, sleep quality improved significantly, as the median Pittsburgh Sleep Quality Index score fell from 9 to 6. Patients with idiopathic pulmonary ...

While further testing is needed in a larger sample of patients, spesolimab may be a promising option for the treatment of combined GPP and PPP. Spesolimab, an interleukin-36 (IL-36) receptor ...

Premanifest individuals showed a selective decline in action initiation, whereas participants with manifest HD exhibited broader, more global increases in apathy. In Huntington disease (HD), apathy is ...

The model had an area under the receiver operating characteristic curve of 0.83 in a high-risk subgroup of patients with conditions that mimic ATTR-CM. An artificial intelligence model trained to ...

Among the most concrete recommendations is a call to establish fit-for-purpose, tiered validation pathways for clinical outcome assessments and biomarkers. A new report from the Reagan-Udall ...

In a phase 3 trial, participants taking venglustat demonstrated statistically significant improvement in neurological symptoms compared with those receiving ERT. Sanofi announced that the US Food and ...

Despite a phase 3 trial that narrowly missed its primary endpoint, Sarepta is pursuing traditional approval for the 2 DMD therapies. Sarepta Therapeutics will submit supplemental new drug applications ...

Upper-to-lower body segment ratio decreased significantly from baseline relative to untreated comparators in both age groups. Continuous treatment with vosoritide in children with achondroplasia shows ...

Tryptophan-kynurenine and arginine-polyamine pathways were strongly linked to severe neurotoxicity and poorer outcomes. Metabolomic pathway-based scores outperformed traditional inflammatory protein ...