New data on Spinraza show benefits in SMA children

New Spinraza (nusinersen) data has been unveiled, which demonstrates benefits for both infantile- and later-onset spinal muscular atrophy (SMA) populations, including improvement in motor function as well as increased survival for the most severely affected, US biotech major Biogen (Nasdaq: BIIB) announced today.

These findings are based on interim results from the SHINE open-label extension study and an analysis of Spinraza’s effects on mobility and fatigability in later-onset participants from the CS2/CS12 studies. The data will be presented at the American Academy of Neurology (AAN) Annual Meeting), currently taking place in Los Angeles.

“These results reinforce Spinraza’s unprecedented and compelling efficacy across a broad range of SMA populations, enabling patients to improve mobility and motor function – and, for the most severely affected, increase their chances of survival,” said Alfred Sandrock, executive vice president and chief medical officer at Biogen, adding: “We look forward to continuing to work with healthcare providers, institutions and SMA communities to provide access to Spinraza for those in need, no matter their age, disease severity or duration of the disease.”