Bulbar involvement improved with Zolgensma in type I SMA

A multidisciplinary American team has carried out post-hoc analyses of the results of the START (phase I) and STR1VE-EU and STR1VE-US (phase III) trials of Zolgensma®.

• They involved a total of 65 infants with type I SMA, followed between 18 and 24 months.
• Following treatment, 92% of them had normal swallowing, 75% were able to feed orally and 92% had no respiratory impairment.
• Of 20 patients, 19 (95%) were able to produce at least two different vowels (the communication criterion in this analysis) and 15 (75%) had improved in the four areas studied (feeding, swallowing, communication and breathing).

Unlike Spinraza® (nusinersen), which appears to have little effect on bulbar functions, Zogensma® is also effective in these areas.

 

Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment. McGrattan KE, Shell RD, Hurst-Davis R et al. J Neuromuscul Dis. 2023.