SENS study to assess feasibility, effectiveness and family experience of introducing SMA screening into the NHS newborn screening programme
The new regimen, delivering 50 mg/5 mL and 28 mg/5 mL doses, offers a higher concentration of the drug for both loading and maintenance phases
The Court dismissed Swiss drugmaker Roche’s plea for interim injunction
Simplified storage and administration of new tablet formulation may provide greater freedom and independence for people with Spinal Muscular Atrophy (SMA)
Evrysdi is the only non-invasive disease-modifying SMA treatment and is approved in over 100 countries
Evrysdi is approved in 70 countries and submitted in a further 31 with more than 4,500 patients treated to date
Biogen paid Ionis a US $ 60 million one-time upfront payment
Salanersen was generally well-tolerated, slowed neurodegeneration, and enabled functional gains
Industry leaders highlighted the pharmaceutical sector’s role in advancing research, innovation, early screening, and multi-stakeholder collaboration
Itvisma is a one-time, fixed-dose therapy designed to tackle the genetic root cause of SMA
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