Tag: spinal muscular atrophy
Newborn screening for SMA leads to more children being able to...
Newborn screening (NBS) for spinal muscular atrophy (SMA), when combined with early treatment, results in better movement ability in affected children, including the ability...
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...
Evrysdi approved in Japan for the treatment of spinal muscular atrophy
PTC Therapeutics has announced that Evrysdi (risdiplam) has been approved in Japan by the Japanese Ministry of Health, Labor and Welfare for the treatment of spinal...
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...
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...
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...