Risdiplam is a new therapeutic agent developed to treat spinal muscular atrophy (SMA), a genetic neurodegenerative disease caused by mutations in the SMN1 gene. Unlike previous invasive therapies, risdiplam offers the ...
Nusinersen, an antisense oligonucleotide, is an active ingredient of the first drug approved by the Food and Drug Administration for the treatment of spinal muscular atrophy, a genetic condition characterized by progressive ...
