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…

$140,000 Grant to Jocelyn Côté, PhD

Cure SMA Canada Awards $140,000 Grant to Joceyln Côté, PhD, University of Ottawa Cure SMA Canada has awarded a $140,000 research grant to Joceyln Côté, PhD, at The University of Ottawa, for his project, “Investigating the mechanism by which SMN regulates translation: identification of novel therapeutic targets.” Understanding the function of the SMN protein is…

SMA Family Camp

Fun….that’s the word that firstly describes SMA Family Camp. The next words would be support, connections, growth and freedom. You may wonder why freedom would be included in the description. Simply put, it’s the freedom you feel when you are in a group setting, surrounded by people that truly “get” what your family life is…

First ever therapy for SMA

Spinraza became the first ever approved treatment for SMA in the USA in December 2016. Spinraza became the first ever approved treatment for SMA in the USA in December 2016. Spinraza became the first ever approved treatment for SMA in the USA in December 2016. Spinraza became the first ever approved treatment for SMA in the USA…