Patients with infantile onset SMA treated with the small-molecule SMN upregulator RG7916 also demonstrated significant gains in function, with 57% of babies achieving CHOP-INTEND (Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders) scores of ≥40 (out of a maximal score of 64), a milestone essentially never reached in untreated SMA Type 1 patients (34). Here, SMN1 is linked to proximal spinal muscular atrophy.