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 ...

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 ...

In rare disease medicine, time is never neutral. For children with thymidine kinase 2 deficiency (TK2d), time can make the difference between irreversible decline and meaningful intervention. My son’s ...

Patient-reported outcomes reflected strong acceptance, with a median satisfaction score of 8.9 out of 10 on the Self-Injection Assessment Questionnaire. The zilucoplan (Zilbrysq ®) auto-injector ...

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 ...

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 ...

Successful treatment involving spesolimab rapid induction and secukinumab maintenance therapy was reported recently in a 62-year-old female patient with annular GPP. In patients with annular ...

Preclinical findings show DYNE-401 enables lower and less frequent dosing while targeting both skeletal muscle and the CNS in Pompe disease. Even when administered every other month, DYNE-401 ...