Efficacy analyses for the first 2 cohorts of the phase 1/2 studies will compare participant outcomes with those of a propensity score-matched external control group. AMT-130, an investigational gene ...

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 ...

Nephrologist Gates Colbert, MD, speaks at the Renal Physicians Association 2026 Annual Meeting in Atlanta, Georgia (Photo by Riya Ajmera) With 4 pivotal therapy types now available, CKD remission is ...

If you know anything about multiple sclerosis (MS), you know that researchers believe there’s a connection between MS and the Epstein-Barr virus (EBV), one of the world’s most common viruses, ...

Peripheral oxygen saturation recovery time and a modified gender-age-physiology index could predict the risk of overall adverse events in patients with IPF. Incorporating peripheral oxygen saturation ...

Median albumin levels rose from 4.2 to 4.7 mg/dL in the efgartigimod group and decreased from 4.4 to 4.0 mg/dL in the rozanolixizumab group. In a single-center observational study of patients with ...

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 ...

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 ...

The adjusted odds ratio for Alzheimer disease among people diagnosed with ATTR-CM was 0.65, suggesting a lower likelihood of developing the disease compared with controls. Transthyretin-mediated ...