SMA12d
Spinraza treatment won’t lead to kidney dysfunction, per study
Spinraza treatment does not lead to significant kidney dysfunction in SMA patients with types 1 and 2, a safety and efficacy ...
Nature3y
The expanding frontier of nucleic acid therapeutics
Nusinersen is a potent example ... a carbohydrate called GalNAc. The molecule binds to a receptor expressed near-exclusively in liver cells. GalNAc-coupled siRNAs efficiently reach that tissue ...
Nasdaq1mon
Biogen Inc. Announces FDA and EMA Acceptance of Higher Dose Regimen for Nusinersen in Spinal Muscular Atrophy
Biogen Inc. announced that the FDA has accepted and the EMA has validated its supplemental New Drug Application for a higher dose regimen of nusinersen, intended for treating spinal muscular ...
Nasdaq1mon
FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA
Applications are based on data from the DEVOTE study, which demonstrate the potential for the investigational higher dose regimen of nusinersen to advance the treatment of SMA CAMBRIDGE ...