Biogen announced new data from the Phase 3 ENDEAR study that showed a statistically significant reduction in the risk of death or permanent ventilation in Spinraza (nusinersen)-treated infants with spinal muscular atrophy (SMA) vs. untreated infants. Spinraza, an antisense oligonucleotide, treats SMA by causing mutations in the chromosome 5q that leads to SMN protein deficiency. It increases production of full-length SMN protein by altering the splicing of SMN2 pre-mRNA.

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