Update from Dr. Rashmi Kothary

Cure SMA Canada Funded Canadian Researcher Dr. Rashmi Kothar Ottawa Hospital Research Institute Biosketch: Dr. Kothary is the Deputy Scientific Director and Senior Scientist at the Ottawa Hospital Research Institute (OHRI). He received a Ph.D. in Biochemistry from the University of British Columbia and pursued postdoctoral research in the laboratories of Dr. Janet Rossant at…

2016 research news

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…

February 5, 2015 Project update for FSMA Canada (Cure SMA) Grant: KT1415 (Feb 1, 2014 to Jan 31, 2016) $50,000 per year

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…

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

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.…

Clinical trial 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 News

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…