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 ...

In MG, patients should typically receive vaccinations before initiating complement inhibitor therapy to avoid unnecessary antibiotic exposure. Experts in Germany, Austria, and Switzerland issued ...

In the ADAPT SERON trial, treatment with efgartigimod led to rapid, significant, and sustained improvements in symptoms such as speech, vision, swallowing, and physical function. The US Food and Drug ...

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 ...

Exploratory outcomes indicated further benefits, including lower rates of myasthenic crises and hospitalizations, as well as reduced need for rescue therapy. Cemdisiran, a small interfering RNA (siRNA ...

Dana Rizk, MD, professor of medicine in nephrology at the University of Alabama at Birmingham Heersink School of Medicine, speaks at the Renal Physicians Association 2026 Annual Meeting in Atlanta, ...

Although 94% of HCPs agreed that transparent communication improves their understanding of patient preferences, only 57% of patient advocacy group representatives concurred. Physicians and patient ...

The rate of progression to end-stage renal disease in patients with nephrotic syndrome is 10% to 20% at 5 years and 30% to 50% at 10 years. 2 The 7-year survival rate of patients with FSGS is 69%, ...

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 ...

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

Benefit with luspatercept persisted in SF3B1-mutated and wild-type disease and at higher variant allele frequencies. Luspatercept demonstrated superior red blood cell transfusion independence and ...