Spinraza: New ENDEAR study results in The New England Journal of Medicine

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy Nusinersen is an antisense oligonucleotide drug that modulates pre–messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA). “The data published in The New England Journal of Medicine further emphasize the benefit SPINRAZA … Fortsätt läsa Spinraza: New ENDEAR study results in The New England Journal of Medicine

New data from Phase 3 ENDEAR study demonstrated earlier initiation of treatment with SPINRAZA may improve motor function outcomes in infants with Spinal Muscular Atrophy (SMA)

Phase 2 EMBRACE interim analysis showed greater motor milestone achievement in infants and children treated with SPINRAZA, compared to those untreated, in patient populations not studied in the pivotal trials Interim analysis of EMBRACE also supported the dosing regimen of four loading doses in the first two months, followed by the administration of SPINRAZA every … Fortsätt läsa New data from Phase 3 ENDEAR study demonstrated earlier initiation of treatment with SPINRAZA may improve motor function outcomes in infants with Spinal Muscular Atrophy (SMA)

New Data to the SHINE open-label extension study

Biogen releases new data from the Shine study (NCT02594124). The has enrolled 292 patients (infants through teenagers) from five previous SPINRAZA clinical studies, including ENDEAR. New findings from the SHINE study show treatment with SPINRAZA resulted in motor function improvement or disease stabilization in toddlers, children and young adults who were treated continuously, some for … Fortsätt läsa New Data to the SHINE open-label extension study