Mifepristone, a glucocorticoid blocker, increased survival and muscle size in a mouse model of spinal muscular atrophy (SMA), according…
Articles by Andrea Lobo
Treatment with Zolgensma (onasemnogene abeparvovec-xioi) improved motor function in children with spinal muscular atrophy (SMA) who needed tracheostomy,…
The specialty pharmacy Orsini was selected as a partner to provide Itvisma (onasemnogene abeparvovec-brve), the newly approved gene therapy…
A high-dose regimen of nusinersen — the active ingredient in Spinraza — has been recommended for approval to treat…
Blocking a small RNA molecule called miR-140-3b can improve motor function in a mouse model of spinal muscular atrophy…
An investigational gene therapy that targets nerve cells thats’s being developed by Exegenesis Bio was well tolerated and improved motor…
Biogen is very pleased with the “striking” results seen thus far in clinical testing with its therapy candidate salanersen…
A higher-dose regimen of nusinersen, currently up for approval in the U.S., may provide further benefits to people with…
Treatment with Evrysdi (risdiplam) is generally safe and effective at improving or stabilizing motor and lung function in adults…
Interim results from a Phase 1 clinical trial show salanersen, Biogen’s treatment candidate for spinal muscular atrophy (SMA),…