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 Update – Sept 2016 – Nusinersin Biogen ENDEAR Update – transition to expanded access program. “… we have reported positive results from a…
Title: The Non-SMN Mediated Benefits of The HDAC Inhibitor Trichostatin A PI: Rashmi Kothary We have just completed the first year of funding from FSMA Canada. Objective: Our goal was to better understand how a small molecule (TSA) that is a global gene regulator ameliorates the disease symptoms and pathology in a mouse model of…
Objective: We will study and obtain more information about the role of a novel protein, called PRMT8, which is present at the surface of the cells that are most affected in SMA, the motor neurons in lower spinal cord. Research Strategy: We will test the possibility that PRMT8 may be able to make the SMN…
CARLSBAD, Calif., June 11, 2015 /PRNewswire/ — Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today provided an update on its ongoing open-label Phase 2 clinical study of ISIS-SMNRx in infants with Type I spinal muscular atrophy (SMA). Previously the company reported data from this study on event-free survival, measures of muscle function and assessments of developmental milestones.…
Identification and Characterization of Factors Critical in Regulating the Selective Vulnerability of Distinct Motor neuron Pools in Spinal Muscular Atrophy Models of Mice 2013
This study will be recruiting shortly, and is open to qualifying Type 1 Infants. Canadian location is Toronto Hospital for Sick Children. From Cure SMA-US: ISIS Pharmaceuticals Initiates Clinical Study in Infants with Spinal Muscular Atrophy. (April 23, 2013) Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced the initiation of a Phase 2 study of ISIS-SMNRx in…
Canadian SMA Research News2012 Cure SMA Canada is proud to have offered major support to the Canadian Neuromuscular Disease Registry in support of SMA registration. This registration will make a substantial impact on standardizing care for SMA across Canada. It will also help initiate the offer of clinical trials in Canada as well as supplying…