New data from Biogen reveals higher Spinraza doses may enhance motor function in SMA patients. Read more here.

Panelists discuss how intrathecal delivery of onasemnogene abeparvovec in the STEER study demonstrates statistically ...

My interest in motor neuron disease was sparked by Jørgen, who has been researching and developing treatments for neurodegenerative patients, including those with MND for over a decade.

Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal muscular atrophy (SMA) in Germany at the start of July priced at 1,945,000 euros, and ...

After all the anticipation, Novartis has the FDA approval it has been seeking for Zolgensma, its gene therapy for spinal muscular atrophy, and has priced it at the low end of expectations. The FDA ...

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Treatment with salanersen slowed neurodegeneration and improved motor function in children with SMA in a Phase 1 trial, interim results show.

Hereditary proximal spinal muscular atrophy (SMA) is caused by survival motor neuron (SMN) protein deficiency due to homozygous loss of SMN1 gene function. Residual SMN protein levels are produced by ...