New high-dose Spinraza moves forward after positive EU opinion
A high-dose regimen of nusinersen — the active ingredient in Spinraza — has been recommended for approval to treat…
Articles by Andrea Lobo
Blocking small molecule improves motor symptoms in SMA mice
Blocking a small RNA molecule called miR-140-3b can improve motor function in a mouse model of spinal muscular atrophy…
SMA gene therapy being tested in mice improves their motor function
An investigational gene therapy that targets nerve cells thats’s being developed by Exegenesis Bio was well tolerated and improved motor…
Biogen moving ‘rapidly’ in quest to bring salanersen to SMA patients
Biogen is very pleased with the “striking” results seen thus far in clinical testing with its therapy candidate salanersen…
Higher-dose nusinersen may further improve SMA outcomes: Developer
A higher-dose regimen of nusinersen, currently up for approval in the U.S., may provide further benefits to people with…
Evrysdi treatment safe, effective in adults with SMA: Real-world study
Treatment with Evrysdi (risdiplam) is generally safe and effective at improving or stabilizing motor and lung function in adults…
Treatment with salanersen shows benefits for SMA children in trial
Interim results from a Phase 1 clinical trial show salanersen, Biogen’s treatment candidate for spinal muscular atrophy (SMA),…
Ketoacidosis not linked to diabetes in woman with SMA type 2
The case of a woman with spinal muscular atrophy (SMA) type 2 who developed an abnormal accumulation of molecules…
Wearable robotic device may boost walking ability for SMA patients
Training with a wearable robotic device called HAL, for hybrid assistive limb, improved walking ability for people with spinal…
SMA Europe’s inaugural gathering to discuss medical emergencies
SMA Europe will host its 1st International Clinical Care Symposium on SMA and focus on the biological causes…