Medical University of Vienna

Gene therapy lenadogene nolparvovec for LHON due to MT-ND4 gene variant showed sustained BCVA improvement in both eyes up to 5 years post-treatment, with 66.1% of patients improving at least -0.3 logMAR (+3 lines) in at least one eye. Vision benefits persisted over 3 years, with no permanent complications and manageable inflammation. The therapy aims to correct the genetic defect causing LHON, a rare inherited optic nerve disease affecting one in 50,000 people.

Abrocitinib shows rapid improvements in atopic dermatitis severity, maintained up to 72 weeks in patients for whom approved systemic drugs were inadequate. The JADE REAL analysis, a multicenter, open-label, expanded access protocol, evaluated 277 participants. Significant reductions in EASI, PP-NRS, %BSA, and POEM scores were observed by 72 weeks, with 78% reporting TEAEs, mostly mild or moderate.

Rising ACR20 response rates in placebo arms of rheumatoid arthritis trials may be linked to global recruitment shifts, with recent trials including more patients from less affluent countries.

Researchers from ISTA and Medical University of Vienna studied human hippocampal CA3, finding it differs from mouse models with sparser neuronal connectivity and more reliable synapses, enhancing memory storage and retrieval. Their findings, published in Cell, highlight the unique features of the human brain.

AutoPiX, funded by the EU and industry with €21 million, aims to use AI for arthritis imaging biomarkers, improving diagnosis, therapy monitoring, and prediction. The project includes automated image analysis, mobile apps for remote patient monitoring, and innovative imaging techniques to replace invasive diagnostics. Coordinated by MedUni Vienna and Janssen, it involves 17 partners from the EU, Switzerland, and the USA.

The RANK/RANKL system drives intestinal adaptation during pregnancy and lactation, enhancing nutrient uptake for mother and offspring, and potentially impacting intestinal cancer and regeneration.

A phase II pilot trial tested very low doses of rituximab in AIHA patients, finding transient CD20+ cell depletion but ineffective sustained suppression. The EC95% of 0.4µg/mL rituximab may guide future low-dose studies.

EC approves Kisqali® (ribociclib) plus AI for HR+/HER2- early breast cancer, based on Phase III NATALEE trial showing 25.1% reduction in recurrence risk. Approval extends eligibility to more patients, addressing high risk of recurrence despite endocrine therapy.

The European Commission approved ribociclib (Kisqali) plus an aromatase inhibitor for adjuvant treatment of hormone receptor–positive, HER2-negative early breast cancer at high risk of recurrence, based on NATALEE trial data showing a 25% reduction in risk of invasive disease, recurrence, or death. The 3-year invasive disease–free survival rate was 90.4% with ribociclib vs 87.1% with AI alone.

EC approves Kisqali (ribociclib) plus AI for HR+/HER2- EBC at high risk of recurrence, based on NATALEE trial results showing 25.1% reduction in disease recurrence risk with Kisqali plus ET vs. ET alone, consistent across subgroups.