Tag: spinal muscular atrophy

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...
NURTURE

Spinal Muscular Atrophy drug may be effective if started later than...

Nusinersen (Biogen) has previously been shown to be effective in the treatment of spinal muscular atrophy in infants under seven months of age; recent...

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...
Mouse

Positive pre-clinical results for Scholar Rock’s SRK-015 therapy in spinal cord...

Data presented at the 35th Annual National Neurotrauma Symposium (Neurotrauma 2017; 9–12 July, Snowbird, USA) have demonstrated beneficial effects for SRK-015 antibody therapy (Scholar...

Informative biomarkers for tracking SMN-modulating spinal muscular atrophy therapies remain elusive

As promising new therapies such as those directly targeting survivor motor neuron (SMN) are entering clinical trials for infants, children, and adults with SMA,...

First spinal muscular atrophy treatment to be submitted for US FDA...

Nusinersen, an investigational treatment, has been shown to significantly improve achievement of motor milestones in babies with infantile-onset spinal muscular atrophy, according to an...