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 ...

A new capsid improves skeletal and cardiac muscle tropism, reducing off-target effects and enabling rapid transduction and transgene expression. New strategies to overcome immune responses against ...

In this case, avacopan monotherapy yielded a sustained AAV remission lasting for over 6 months, even in the absence of steroid treatment. Avacopan can induce sustained, steroid-free remission in ...

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 ...

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 ...

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 ...

Results show that it may be beneficial to add longitudinal monitoring of kidney function to the management of patients with MDS. Myelodysplastic syndromes (MDS) are associated with a substantially ...

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

Survival outcomes were similar with hypomethylating agents (HMAs) alone and an HMA plus venetoclax despite higher overall response rates. Investigators retrospectively evaluated 140 patients with TP53 ...

Spesolimab is a humanized monoclonal immunoglobulin G1 antibody and is the first therapy to target the IL-36 pathway, a key driver of GPP disease processes. Spesolimab has an acceptable safety profile ...