Change in HFMSE score from baseline was strongly correlated with change from baseline in ULM score at every visit day in patients with Type 2 SMA. Progressive improvements in mean ULM score were demonstrated over time (4.0-point increase from baseline).1
HFMSE=Hammersmith Functional Motor Scale—Expanded; SE=standard error; SMA=spinal muscular atrophy; ULM=Upper Limb Module.
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
1. Darras BT, Chiriboga CA, Iannaccone ST, et al; on behalf of the ISIS-396443-CS2/ISIS-396443-CS12 Study Groups. Nusinersen in later-onset spinal muscular atrophy: long-term results from the phase 1/2 studies [published online April 24, 2019]. Neurology. doi:10.1212/WNL.0000000000007527.
2. Kaufmann P, McDermott MP, Darras BT, et al; Muscle Study Group (MSG); Pediatric Neuromuscular Clinical Research Network for Spinal Muscular Atrophy (PNCR). Prospective cohort study of spinal muscular atrophy types 2 and 3. Neurology. 2012;79(18):1889-1897.