Tag: SMA

Risdiplam associated with significant improvements in motor function in type 2...

The use of once-daily risdiplamin in type 2 and non-ambulant type 3 spinal muscular atrophy (SMA) patients leads to a significant improvement in motor...

Subcutaneous intrathecal catheter provides reliable administration of nusinersen for SMA patients

For spinal muscular atrophy (SMA) patients with complex spine anatomy, the subcutaneous intrathecal catheter (SIC) allows for reliable outpatient administration of nusinersen that results...

Significant improvements in survival and developmental milestones for Type 1 SMA...

Symptomatic Type 1 spinal muscular atrophy (SMA) infants treated with Evrysdi (risdiplam) showed significant improvements in survival, motor milestones, and motor function compared to...

Biogen scoops sixth Prix Galien award with UK for life-changing rare...

Biogen was announced as the winner of the Orphan Product Award at last night’s UK Prix Galien 2018, for Spinraza, which in 2017 became...
NURTURE

New data show Spinraza beneficial in the treatment of spinal muscular...

Rates of survival are higher, and respiratory intervention rates are lower, in 25 infants with presymptomatic 5q spinal muscular atrophy (SMA) treated with Spinraza...

Preliminary study suggests drug may help infants with Spinal Muscular Atrophy

A preliminary study suggests that an investigational drug may help increase protein levels in infants with spinal muscular atrophy. The results of the open label study were...