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.…