The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

SMA Europe

The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

Latest News

13/01/2016

Ionis Pharmaceuticals completes enrollment for Phase 3 clinical trial of Ionis-SMNrx in children

Ionis Pharmaceuticals announced today that it has earned a milestone payment of $2.15 million from Biogen for completing the target enrollment of the Phase 3 CHERISH study. The study is designed to support marketing approval of Ionis-SMNrx in children with spinal muscular atrophy (SMA). B. Lynne Parshall, chief operating officer at Ionis Pharmaceuticals, said: “Achieving our […]

07/01/2016

Roche announces new clinical trial for RG7916 in spinal muscular atrophy

Roche, along with partners PTC Therapeutics and the SMA Foundation in the US, recently provided an update on the development of RG7800 and announced a new clinical trial planned for RG7916. The study will take place in The Netherlands. RG7916 is another SMN2 splicing modifier. This new investigational medicine will now advance to its very first clinical […]

07/01/2016

AveXis completes phase 1 clinical trial enrollment

AveXis has announced that they have completed enrollment in a Phase 1 study testing systemic delivery of gene therapy product AVXS-101, for spinal muscular atrophy. The open-label, dose-escalation study is designed to evaluate safety and preliminary indications of efficacy of AVXS-101 in patients with SMA Type 1. The primary outcome in the study is safety and […]

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