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 ...

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 ...

Between 1970 and 1995, the incidence of new NHL cases increased. Between 2007 and 2016, new cases declined by approximately 0.9% per year, and mortality from NHL decreased by 2.2% per year. 3 ...

Older age and higher-risk protocols significantly increased odds of nutritional deterioration. Children undergoing induction chemotherapy for acute lymphoblastic leukemia (ALL) frequently experienced ...

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 ...

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

The CAPTIVATE trial met its target of at least 20 responders among the first 40 participants before all 40 completed part A. Dianthus Therapeutics announced an early “go” decision for claseprubart for ...

The antibodies were slightly more common in double seronegative MG, at 3.9%, than in seropositive MG, at 1%. In patients with suspected myasthenia gravis (MG), LRP4-IgG antibodies are rare, with poor ...

Outcomes improved in patients who received nomlabofusp, while scores worsened in a matched reference cohort from the FACOMS natural history database. Larimar Therapeutics announced that the US Food ...

Consolidation or maintenance therapy significantly improved progression-free survival and overall survival. Zanubrutinib, lenalidomide, temozolomide, and rituximab (RLZT) combined with or without high ...

Wild-type ATTR-CM comprised 76% of cardiac amyloidosis cases, with prevalence highest in Asia and lowest in North America. Cardiac amyloidosis (CA) is present in approximately 10% of patients with ...

If approved, CAM2029 could offer a self-administered alternative to intramuscular long-acting somatostatin analogs, with the potential to reduce treatment burden. The US Food and Drug Administration ...