Author Archives: Margarida Maia

FDA Clears Way for KIK-AS Phase 1/2 Trial to Resume

The U.S. Food and Drug Administration (FDA) has lifted the clinical hold on a Phase 1/2 study testing GTX-102 — an investigational therapy by GeneTx Biotherapeutics and Ultragenyx Pharmaceutical — in children and adolescents with Angelman syndrome. GeneTx is now cleared to begin dosing treatment-naïve patients — those who have never received…

Neuren Closer to Phase 2 Testing of NNZ-2591 After FDA Guidance

Neuren Pharmaceuticals is a step closer to Phase 2 clinical testing of NNZ-2591, its investigational therapy for Angelman syndrome, Phelan–McDermid syndrome, and Pitt Hopkins syndrome, after the company received clear and constructive guidance from the U.S. Food and Drug Administration. All three syndromes are early…