Ultragenyx Pharmaceuticals' apazunersen did not meet the goals of its Phase 3 test in Angelman syndrome, a rare genetic neurological disorder with no FDA-approved therapies. The failure of what was once considered a potential blockbuster therapy is forcing Ultragenyx to examine significant cost cuts.
Novartis temporarily halted enrollment and treatment in eight clinical trials of an experimental CAR-T therapy for autoimmune and neurological disorders in late August after three patients died. Bristol Myers Squibb programs were also paused.
Researchers have developed a new interventional treatment for hepatic myelopathy, a rare neurological complication of chronic liver disease that previously could only be cured through liver transplantation.
Kyverna Therapeutics announced plans to submit its one-time CAR-T cell therapy for stiff person syndrome to the FDA after late-stage trial showed improvements in mobility and reduced disabilities in patients with the rare neurological disease.
