Ionis stock rises on FDA, EMA nod for SMA drug dose increase
Shares of Ionis Pharmaceuticals (NASDAQ:IONS) climbed 7% today after the company announced that regulatory agencies in the United States and Europe have accepted applications for a higher dose regimen ...
Nasdaq18d
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 ...
Nature1y
The translational gap: bridging basic research, clinical practices and society
The approval of nusinersen, an RNA-based drug that restores muscle function when injected into the spine, marked a significant milestone in the field of gene therapy. Since then, thousands of ...
Nature3y
The expanding frontier of nucleic acid therapeutics
For example, Biogen’s nusinersen, an ASO therapy for the fatal neuromuscular condition spinal muscular atrophy (SMA), can reduce by half the likelihood of death or the need for mechanical ...
SMA7d
COVID-19 made life with SMA more difficult, parents and doctors say
In surveys, parents of children with SMA and healthcare professionals in Sweden note the many ways the pandemic affected ...
Toronto Star19d
FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA
The higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and higher maintenance regimen, 28 mg, every 4 months, compared to the approved nusiner ...
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 SMACAMBRIDGE, Mass., Jan. 23 ...