Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an antisense oligonucleotide d ...

Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as treatments for ...

Findings from an Italian cohort suggest that nusinersen may benefit fatigue measures in patients with spinal muscular atrophy (SMA) type III; however, drawing definitive conclusions in this area ...

Nusinersen in SMA: indication of major added benefit in children with early-onset disease Treatment delays permanent ventilation of the children and prolongs life. In addition, the children ...

New analyses from DEVOTE Part C further characterize the improvements in motor function in participants with SMA who transitioned to the investigational higher dose regimen of nusinersen from 12 ...

New analyses from DEVOTE Part C further characterize the improvements in motor function in participants with SMA who ...

Researchers of the multicenter study looked at the effect of nusinersen among 48 patients with spinal muscular atrophy (SMA) from 2 Italian hospitals, finding more active immune systems among the ...

Biogen said that new data examining of its already approved spinal muscular atrophy treatment Spinraza (nusinersen) indicates ...

Findings from Part B and Part C of the DEVOTE study support the clinical benefits of a higher dose regimen of nusinersen (50/28 mg) in both individuals previously treated and treatment-naïve to ...

A positive effect of nusinersen treatment on motor function in ambulant pediatric and adult spinal muscular atrophy (SMA) patients during a 38-month period has been demonstrated by an analysis of ...

--Ionis Pharmaceuticals today announced that its partner Biogen presented new data highlighting the potential benefit of SPINRAZA ® in infants and toddlers living with spinal muscular atrophy ...

The nusinersen clinical development program encompasses more than 10 clinical studies, which have included more than 460 individuals across a broad spectrum of patient populations, including two ...