FDA approves Spinraza (nursinersin) for treating SMA – Dec 2016. Canadian locations were part of the clinical trials. There are many more details at the Cure SMA (USA) research review page.
Biogen ENDEAR Update – transition to expanded access program. “… we have reported positive results from a pre-specified interim analysis of the ENDEAR study in infants with infantile-onset Spinal Muscular Atrophy (consistent with Type 1). As a result, plans are underway to transition all ENDEAR study participants to nusinersen in an open-label extension study and close the ENDEAR study. We plan to submit regulatory filings globally in the coming months. This step will hopefully bring us closer to our ultimate goal of making nusinersen available as quickly as possible as an approved therapy.” (Sept 2016)
Research Update from Roche – SMN2 Splicing Modifier Programme (Sept 2016)
Cure SMA Canada has awarded a $140,000 research grant to Rashmi Kothary, MD, at the Ottawa Hospital Research Institute for his project, ” Muscle satellite cell biology and muscle regeneration in Smn-depleted mice.” (Feb 2016)
Ionis (formerly ISIS) Study, “A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy.” – clinical trial ongoing but recruiting now closed. (Sept 2016) Locations are Toronto, Montreal and Vancouver