#Biogen and #IonisPharmaceuticals Report #Nusinersen Meets Primary Endpoint at Interim Analysis of Phase 3 ENDEAR Study in Infantile-Onset #SpinalMuscularAtrophy – #SMA

Originally posted 2016-08-02 11:05:12.

Biogen (NASDAQ:BIIB) and Ionis Pharmaceuticals (NASDAQ:IONS) today announced that nusinersen, their investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint pre-specified for the interim analysis of ENDEAR, the Phase 3 trial evaluating nusinersen in infantile-onset (consistent with Type 1) SMA. The analysis found that infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones compared to those who did not receive treatment. Nusinersen demonstrated an acceptable safety profile in the trial.

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