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Page last checked: 14th December 2022
Last updated: 21st February 2022


The CHERISH trial¹ was with children with SMA Type 2 aged 2-12 years. Participants had to be able to sit independently and have no history of the ability to walk independently. They also couldn’t take part if they needed help with breathing, had significant curvature of the spine, or were fed via a gastric tube. 126 children were screened and enrolled with the trial; the youngest child was aged 2 years, the oldest child was aged 9 years.

The trial was set up to assess how children treated intrathecally with nusinersen via a lumbar puncture (see section “How is the treatment delivered?”) on days 1, 29 and 85, and with a maintenance dose on day 274, compared to those having a sham procedure – a small needle prick to the lower back, which was covered to simulate the appearance of a lumbar puncture. Children and families didn’t know whether they were receiving the nusinersen or sham treatment (a double-blind study). The assessment of the impact (or outcome) of the treatment was to be primarily measured using a physiotherapy assessment tool called the Hammersmith Functional Motor Scale-Expanded (HFMSE)²’³.

Two thirds (84) of the children received nusinersen treatment; 42 received the sham procedure. The first child underwent the first part of the procedure on November 24th 2014, and the last child’s last visit was on February 20th 2017.

The study had set a pre-specified time point of 31st August 2016 for an interim analysis of the results. This was when all children had been enrolled for at least 6 months and at least 39 children had completed the 15-month assessment.

Children who had had full 15 months treatment – observed data Children who had had at least 6 months treatment – multiple imputed data Total
Nusinersen Treatment 35 49 82
Sham Treatment 19 23 42
Total 54 72 126
Table 1. Numbers of children taking part in the Interim Analysis


The interim analysis of these children’s outcome measures confirmed the positive effect of nusinersen. This led to the trial being ended early, as it was considered unethical to continue to give the sham procedure to a group of children when nusinersen was effective at treating symptoms.

All the children who had not had a 15-month assessment were invited to attend a visit that represented the end of the double-blind study period; by this time, some but not all of these children had completed the 15-months treatment schedule. At this visit, all assessments that had been scheduled for the 15-month assessment were performed. The results of all children who accepted this invitation were therefore presented as the final analysis.

Children who had had full 15 months treatment – observed data Children who had had at least 6 months treatment – multiple imputed data Total
Nusinersen Treatment 66 18 84
Sham 34 8 42
Total 100 26 126
Table 2. Numbers of children taking part in the Final Analysis

1. Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, et al. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. New England Journal of Medicine. 2018;378(7):625-35.

2. O’Hagen JM, Glanzman AM, McDermott MP, Ryan PA, Flickinger J, Quigley J, et al. An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients. Neuromuscular Disorders. 2007;17(9):693-7.

3. Murphy A, Mayhew A (2019) Physiotherapy-based measures (HINE, CHOP-INTEND, HMFSE) for people with SMA and how they are used to monitor treatment outcomes (last accessed 13th September 2019)


October​: Poster : Efficacy and Safety of Nusinersen in Children with Later-Onset SMA: End of Study Results from the Phase 3 CHERISH Study presented at the 22nd International Annual Congress of the World Muscle Society (WMS)

April: Biogen presents interim study results at the American Academy of Neurology meeting being held in Boston, USA (April 22nd-28th)


8th November: Biogen announces, ‘due to the positive interim analysis, the CHERISH study will be stopped and participants will be able to transition into the SHINE open-label extension study to receive nusinersen.