The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
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 […]
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 […]
Cytokinetics has initiated enrollment for a Phase 2 study testing CK-2127107 in teens and adults with SMA type II, III or IV, in California, USA. The clinical trial is designed to assess the effect of CK-2127107 on multiple measures of muscle function in both ambulatory and non-ambulatory patients with SMA. In collaboration with Astellas, Cytokinetics is […]
In response to numerous requests from the SMA community, Biogen and Ionis (previously ISIS) Pharmaceuticals, provide an update on the clinical development on their investigational compound, currently being tested in Phase 3 clinical trials. To read the letter, please click here.
Isis Pharmaceuticals, Inc. today announced that the company has changed its name to Ionis Pharmaceuticals, Inc. Ionis Pharmaceuticals is an original name that the Company has chosen to represent its innovative culture and heritage as both the pioneer and leader in the RNA-targeted therapeutic space for the past 26 years. Ionis is focused on bringing […]