Muscle fibers in children with SMA type 1 show substantial differences in components of the myosin protein called heavy ...

More than half of SMA patients in an Australian survey said they did not notice any improvement in their symptoms following ...

while Biogen’s antisense drug Spinraza instructs the body to produce a working version of the SMN protein that is the root cause of the disease. Latest data came from Part 2 of the FIREFISH ...

Researchers report that they have identified a key pathway that sets off neurodegeneration in the early stages of amyotrophic ...

However, by the time this protein aggregation develops ... Interestingly, SmD1 is a survival of motor neuron (SMN) complex ...

In general, each corresponds to the copy number of the SMN2 gene, which produces a small fraction (~10%) of functional SMN protein, essential to the survival of motor neurons, compared with SMN1.

The competitive landscape in SMA treatment is significant, with existing therapies targeting the SMN protein, which may ...