Sezan Mergen Kilic

Long-term real-world outcomes of nusinersen treatment in patients with spinal muscular atrophy types I-III.

BACKGROUND Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder caused by mutations or deletions in the survival motor neuron 1 (SMN) gene, with disease severity influenced by SMN2 copy number. Nusinersen, an antisense oligonucleotide therapy, has emerged as an effective treatment option. This...