Real-world outcomes of spinal muscular atrophy treatment with onasemnogene abeparvovec in Croatia: a comprehensive case series and literature review
Front Med (Lausanne). 2025 Jun 24;12:1609072. doi: 10.3389/fmed.2025.1609072. eCollection 2025. ABSTRACT INTRODUCTION: The development of novel treatment options and the implementation of newborn screening programs …