The first two therapies that received Health Authority (HA) approval for the treatment of SMA were as follows: the intrathecally administered SMN2‐targeting antisense oligonucleotide nusinersen (SPINRAZA®)8 and onasemnogene abeparvovec‐xioi (ZOLGENSMA®), a gene‐transfer therapy that uses a nonreplicating adeno‐associated virus to deliver a functional copy of an SMN1 gene by a single intravenous infusion in patients aged < 2 years.9 Here, SMN2 is linked to proximal spinal muscular atrophy.