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Page checked: 29th November 2022
Last updated: 21st February 2022


The ENDEAR Trial¹’² was with 122 children with SMA Type 1 of whom two-thirds were treated with nusinersen. One third of these received sham treatment – a small needle prick to the skin over the lumbar area of the spine, which was covered with a bandage to simulate the appearance of a lumbar-puncture injection.

At a pre-specified time point in this study, the interim analysis of results showed a significantly higher number of patients in the nusinersen group had a motor-milestone response compared with patients in the sham group. This resulted in the trial being ended early after 13 months and patients from the sham group being moved on to active treatment. It is important to note that when this trial ended some children had completed the trial and had treatment for all 13 months, others were only part-way through.

The results for all the treated children, no matter how long their treatment had been, were that:

  • 51% improved their developmental motor milestones as measured on the HINE scale for assessing motor function compared with 0% of the placebo group. These were the additional skills gained for the treated group:
  • 22% of infants had head control;
  • 10% could roll over;
  • 8% could sit without support;
  • 1% able to stand, compared with 0% of the placebo group.
  • 71% of the treated group made a 4-point gain using the CHOP-INTEND (a second scale that also measures motor function) compared with 3% of the placebo group.
  • 61% of the treated group did not require permanent ventilator support and were still alive. Permanent ventilator support is defined as more than 16 hours non-invasive ventilation support (NIV such as BiPap) for more than 21 days due to the impact of the condition. It does not include those who have to have NIV due to an ongoing infection. This compared with 32% of the placebo group.

1. Servais L, Farra M, Finkel R S, Kirschner J, Muntoni F, Sun P, Gheuens S, Schneider E, Farwell, W (2017) Nusinersen demonstrates greater efficacy with shorter disease duration: final results from the ENDEAR study in infants with Spinal Muscular AtrophyPoster presented at the 22nd International Annual Congress of the World Muscle Society, Saint-Malo, France.

2. Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, Chiriboga CA, Saito K, Servais L, Tizzano E, Topaloglu H, Tulinius M, Montes J, Glanzman AM, Bishop K, Zhong ZJ, Gheuens S, Bennett CF, Schneider E, Farwell W, De Vivo DC; ENDEAR Study Group (2017) Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med 377: 1723-1732.


15th December: Summary and Comparison of Results to date for Nusinersen (ENDEAR Trial) and AVXS-101 Gene Therapy

15th November: Final Results Published: Poster presented at the 22nd International Annual Congress of the World Muscle Society.


15th December: Phase 2 results:

2nd August: Biogen and Ionis Pharmaceuticals announce plan to open an expanded access program (EAP) in the Autumn of 2016, to provide access to patients with infantile onset SMA (consistent with Type 1) prior to potential regulatory approval.