Nusinersen - Other Published Summaries of Treatment Outcomes

Nusinersen is not a cure for SMA Type 1, it's a treatment. Clinical trials only began in 2011, which means that the long-term outcomes are not yet known. In this section, we will bring you summaries of the latest published studies and reviews of outcomes in 'the real world'. We also list other articles you may want to read.

October 2020: Independent Real-World Study Demonstrates Significant Improvements for Adults who have SMA Type 3, Treated with Nusinersen

In response to a request from SMA Europe, Biogen has sent this community update which includes the observation that the most relevant treatment effect was seen in those with SMA Type 3 who were unable to walk without assistance at the start of treatment.

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June 2020: Further small studies support the use of nusinersen for SMA treatment in adults.

Three scientific reports have been published that present observations of adults who have SMA treated with repeated injections of nusinersen into the fluid bathing the spinal cord and brain.

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March 2020: Study Shows Nusinersen is Safe and Effective for SMA Treatment in Adults

A study in a large cohort of adults with SMA Types 1 to 3, indicates that nusinersen is safe and effective at improving muscle function. Published in the Lancet, this study was observational in design and conducted in Germany.

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October 2019: Two New Studies Show Positive Effects of Nusinersen in Later-Onset SMA

Two small, observational studies of the effects of nusinersen in patients with later-onset SMA have recently been published, both of which describe clinically meaningful improvements caused by the treatment. Read more in this article from our Scientific Research Correspondent, Dr James Sleigh:

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May 2019: Nusinersen in Later-Onset Spinal Muscular Atrophy Long-Term Results from the Phase 1/2 Studies  

Darras and colleagues who have recently released information on the longer-term results of the nusinersen trials. The results showed significant differences between treated and untreated groups in their ability to perform tasks over an almost three-year period. This article, from Dr Alex Murphy, our Clinical Care Research Correspondent, summarises their findings.

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December 2018: Current Knowledge and Existing Gaps

Dr Alex Murphy, our Clinical Care Research Correspondent, summaries the article written by Gidaro and Servais, two neuromuscular specialists involved in the clinical trials into SMA treatment, that was accepted for publication in July 2018. It discusses what is currently known, and what aspects of treatment still require research.

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September 2018: Articles reviewing the various 'real world' outcomes of treatment that may be of interest

A number of published articles were used to inform our consultation response to NICE. You can see a list here, along with the quotations we selected. Public access to some of these articles may be restricted.

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