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family handout/Courtesy of the family
The Alberta government has approved funding to treat a rare and often fatal disease in young children with a gene therapy that is considered the world’s most expensive pharmaceutical drug.
Health Minister Tyler Shandro said the province would pay for Zolgensma, which treats Type 1 spinal muscular atrophy, or SMA, on a case-by-case basis while provincial governments negotiate with the drug maker to work out long-term funding. A one-time treatment of Zolgensma costs about US$2.1-million. Alberta is the second province after Ontario to announce such interim funding.
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The global Phase 4 RESPOND study will evaluate the efficacy and safety of SPINRAZA in patients with a suboptimal clinical response to Zolgensma
Clinical and real-world experience have reported that some patients previously treated with Zolgensma have also been treated with SPINRAZA
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Biogen remains committed to exploring the potential of SPINRAZA to optimize outcomes for patients with SMA
CAMBRIDGE, Mass., Jan. 08, 2021(Nasdaq: BIIB) today announced that the first patient has been treated in the global clinical study, RESPOND. The Phase 4 study will examine the clinical benefit and assess the safety of SPINRAZA (nusinersen) in infants and children with spinal muscular atrophy (SMA) who still have unmet clinical needs following treatment with gene therapy Zolgensma