Acadia Pharmaceuticals published real-world data from the LOTUS study demonstrating Daybue® (trofinetide) benefits in Rett syndrome symptom management under routine care. Ionis Pharmaceuticals celebrated 10 years of Surf Away+, an adaptive experience event serving rare neurological disease communities. Arrowhead Pharmaceuticals filed regulatory submissions to initiate clinical trials of ARO-MAPT, an RNAi therapeutic targeting tauopathies including Alzheimer’s disease. These advancements spotlight innovations in diagnosis and treatment for underserved conditions.