Tel: 01789 267520

key clinical trial results

SMA Type 1 (ENDEAR Trial)

In August 2016, Biogen and Ionis announced that nusinersen had met its primary endpoint in an interim analysis of ENDEAR, a double-blind, sham procedure-controlled trial testing nusinersen in infants with SMA Type 1. As a result, the trial was ended early and all participants were transitioned into SHINE, an open-label extension trial (see below).

You can read our key research update summaries here:

  • 15th November 2017

(Poster 3): Nusinersen Demonstrates Greater Efficacy in Infants with Shorter Disease Duration: Final Results from the ENDEAR Study in Infants with SMA

  • 15th December 2016

NUSINERSEN PHASE 2 TRIAL RESULTS PUBLISHED IN THE LANCET

  • 2nd August 2016 

BIOGEN AND IONIS PHARMACEUTICALS' STATEMENT RE NUSINERSEN ANNOUNCEMENT

SMA Types 2-3 (CHERISH Trial)

On November 7 2016, Biogen and Ionis announced that nuisnersen had also met its primary endpoint in an interim analysis of CHERISH, a double-blind, sham procedure-controlled trial testing nusinersen in children with SMA Type 2. As a result, the trial was ended early and all participants were transitioned into SHINE, an open-label extension trial (see below).

You can read our key research update summaries here:

  • 15th November 2017

(Poster 1):
Efficacy and Safety of Nusinersen in Children with Later-Onset SMA: End of Study Results from the Phase 3 CHERISH Study

  • 25th April 2017

Update on Cherish and Nurture clinical trials of spinraza

  • 8th November 2016

BIOGEN AND IONIS PROVIDE COMMUNITY UPDATE ON CHERISH NUSINERSEN TRIAL

For details of the steps towards the development of the drug, read the research updates that were posted between 2011 and 2017. Visit this page and scroll down.

SMA Types 1-3 (SHINE Trial)

SHINE is an open-label, phase 3 study designed to extend the treatment regime of patients who have completed the ENDEAR or CHERISH trials.

You can read our key research update summaries here:

  • 23rd October 2015

ISIS Pharmaceuticals initiates new ISIS-SMNRExtension Study, SHINE

Pre-symptomatic SMA (NURTURE Trial)

NURTURE is a phase 2 trial that aims to evaluate the safety and tolerability of Spinraza in pre-symptomatic newborns that are very likely to develop SMA based on their genetics.

You can read our key research update summaries here:

  • 25th April 2017

Update on CHERISH and NURTURE clinical trials of Spinraza

EMBRACE Study

EMBRACE is a Phase 2 trial of nusinersen that enrolled a small number of SMA patients that were ineligible for inclusion in the ENDEAR and CHERISH Phase 3 trials.

  • 15th November 2017

(Poster 2):
Safety and Efficacy of Nusinersen in Infants/Children with SMA: Part 1 of the Phase 2 EMBRACE Study

To keep up to date with progress on further clinical trial results, you can follow us on Facebook and Twitter and sign up to receive our monthly E-news updates.