Real-world outcomes of spinal muscular atrophy treatment with onasemnogene abeparvovec in Croatia: a comprehensive case series and literature review

Despite the widespread use of novel treatment modalities that have drastically improved patient outcomes, there remains a paucity of real-world case reports documenting the care of SMA patients. This case series and accompanying literature review reinforce the efficacy and safety of onasemnogene abeparvovec in the treatment of SMA type 1, particularly when initiated early. In addition, our case series emphasize the critical role of newborn screening in identifying affected individuals before the onset of irreversible motor neuron loss as well as prompt start of treatment in all patients. We hope that our findings will contribute meaningfully to the expanding body of literature and knowledge on spinal muscular atrophy, ultimately fostering better patient care and outcomes.