Biogen's treatment for neurodegenerative disorder spinal muscular atrophy (SMA) Spinraza has come under pressure in an increasingly competitive market, but the company hopes a new high-dose ...

Apitegromab could achieve blockbuster status with peak revenue potential >$2.5bn, especially based on its ability to build muscle mass in combo with gene therapies like Spinraza and Zolgensma.

The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary ...

In the US Spinraza costs $750,000 for the first year, and $375,000 thereafter, although in the UK the list price is slightly lower at around £450,000 ($581,000) for the first year.

The company provides TECFIDERA, VUMERITY, AVONEX, PLEGRIDY, TYSABRI, and FAMPYRA for multiple sclerosis (MS); SPINRAZA for spinal muscular atrophy; ADUHELM to treat Alzheimer’s disease ...

According to the plaintiffs, Generium violated one of the patents for Spinraza held by the American side. SMA is a rare genetic disease that results in the development of progressive muscle weakness.

Testing patient DNA with advanced methods like HapSMA — which can “[analyze] … SMN and its surrounding genes” — can reveal otherwise undetectable genetic changes, such as gene conversion, that affect ...

Last December, my brother kept badgering me about what I wanted for Christmas. I was so sick with a respiratory infection at the time that my only focus was on getting better, not a present. To my ...