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Canadian SMA Research News

FSMAC has been supporting the best in Canadian SMA research since 2001. Below are some of the current projects we fund.

2016 Research News

Biogen Update - Sept 2016 - Nusinersin

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)

Families of 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

2015 Research News

Project update for FSMA Canada -- The Non‐SMN Mediated Benefits of The HDAC Inhibitor Trichostatin A. PI: Rashmi Kothary - Feb 2015

Arginine Methylation as a Regulator of SMN activities in Motor Neurons - Jocelyn Côté, Ph.D. - Feb 2015


Isis Pharmaceuticals Reports Data from ISIS-SMN Rx Phase 2 Study in Infants with Spinal Muscular Atrophy - June 2015

Identification and Characterization of Factors Critical in Regulating the Selective Vulnerability of Distinct Motor neuron Pools in Spinal Muscular Atrophy Models of Mice - May 6, 2013

Clinical Trial for Type 1 Infants - Toronto Sick Kids - April 26, 2013

Families of SMA Canada funds a New Research Project by Dr. Jocelyn Cote from the University of Ottawa for $140,000 (2013)

Canadian Neuromuscular Disease Registry (CNDR) and SMA (2012)


For American and International SMA research News, go to the FSMA-US Research News section.