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

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

Researchers at UC San Diego identify a key pathway leading to neurodegeneration in early stages of ALS, hinting at the ...

However, by the time this protein aggregation develops, the damage is well underway ... Interestingly, SmD1 is a survival of ...

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.

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 ...