BRIEF—Biogen presents interim results from RESPOND study at Cure SMA

30 June 2023

US biotech major Biogen presented interim clinical outcomes results from the RESPOND study highlighting the potential benefit of Spinraza (nusinersen) in infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with gene therapy Zolgensma (onasemnogene abeparvovec).

The presentation took place at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Orlando, USA.

Of note, interim results at six months from 29 study participants treated with Spinraza show:

Improvements in motor function in most participants as measured by increased mean total Hammersmith Infant Neurological Examination Section 2 (HINE-2) score from baseline.
Most participants (25/27) with investigator-reported suboptimal motor function at baseline improved.
No new emerging safety concerns identified in enrolled participants who received Spinraza after Zolgensma.