“Great news!” was the reaction of one SMA News Today columnist to word of a first gene therapy, Zolgensma, being approved to treat infants and children up to age 2 with spinal muscular atrophy, while others on the site’s SMA community forum called it “an amazing advancement” and “such a…
The National Institute for Health and Care Excellence (NICE), the U.K. agency that decides on the cost-effectiveness of therapies for inclusion in the public health system of England and Wales, is recommending against Spinraza (nusinersen) being offered to treat people with spinal muscular atrophy (SMA). This stance, released as “draft…
AveXis, which is running a series of pivotal clinical trials testing a gene therapy in babies and children with spinal muscular atrophy (SMA), announced plans to be acquired by Novartis for $8.7 billion in cash. The merger, to be completed by mid-year, met with the unanimous approval…
Spinal muscular atrophy (SMA) patients in the European Union will soon have access to Spinraza (nusinersen), the first treatment approved for people with this disease and one able to treat almost all types of SMA. The European Commission announced late Thursday that it had approved Spinraza, marketed by…
Motor milestones — a mark of life quality and potential longevity — are evident in a majority of infants with type 1 spinal muscular atrophy (SMA) treated with Spinraza (nusinersen) in a Phase 3 clinical trial, compared to those who received placebo. New interim trial data, in fact, found that 51 percent of Spinraza-treated infants continue…
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