Nearly all patients in this cohort achieved a clinically meaningful >3-point increase during the study period, demonstrating a consistent response to the gene therapy.
The drug was previously approved for the treatment of acute repetitive seizures and granted 7 years of orphan drug exclusivity in January 2020.
Interim analysis compared ataluren in a real-world setting from the STRIDE registry with a matched cohort; the final data from the STRIDE registry are expected in 2025.
James Leverenz, MD, director of the Lou Ruvo Center for Brain Health at Cleveland Clinic, sat down to discuss the challenges faced in diagnosing Lewy body dementia.
No progressive or delayed complications of treatment were recorded after 3 years in patients with essential tremor who underwent focused ultrasound.
Study data demonstrates that treatment with satralizumab reduces risk of relapses in patients with neuromyelitis optica spectrum disorder.