Researchers found that 16 out of 21 CNGB3 gene variants altered RNA splicing, allowing clearer classification of genetic mutations linked to achromatopsia.
The FDA has approved a HIF-2α inhibitor for adult and pediatric patients aged 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma and paraganglioma (PPGL).
The ASCO Post
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April’s FDA approvals feature first-in-class therapies, targeted biologics, expanded indications, and innovative delivery systems across allergy, endocrinology, rheumatology, oncology, infectious diseases, and more.
Health care facilities face new challenges as sensitive locations policy protecting them from immigration enforcement was rescinded in January 2025, requiring new protocols for patient protection.
The first FcRn blocker for both adult and pediatric generalized myasthenia gravis has been approved by the FDA, based on data showing sustained symptom improvement and IgG reduction in clinical trials.