Investigator Dr. Maryam Oskoui Receives CIHR Grant to Fund Spinal Muscular Atrophy Clinical Trial
NMD4C investigator Dr. Maryam Oskoui has been awarded a Canadian Institutes of Health Research Clinical Trials Operating Grant ($1,777,095) for INFORM SMA as nominated principal applicant. Several NMD4C members are listed on this grant as co-investigators, including Katrina Cardiff, Shaainthabie Karthigesu, Dr. Kathryn Selby, Dr. Jean Mah, Dr. Heran Gonorazky, Dr. Hugh McMillan, Dr. Jordan Sheriko, Dr. Victoria Hodgkinson, and Dr. Lawrence Korngut; and as collaborators, including Niamh Cushen, Catherine Desautels, Dr. James Dowling, Monique Emond, Stacey Lintern, Sarah Turgeon-Desilets, Susi Vander Wyk, and Angelina Woof.
INFORM SMA
INFORM SMA is embedded in the INFORM RARE network, whose vision is to improve health care for children with rare genetic diseases by generating real-world evidence to support decision-making about new and existing therapies. The goal of this trial is to evaluate the effectiveness of a home-based exergame intervention in promoting physical activity for children and youth with Spinal muscular atrophy (SMA) while validating digital outcome measures. Multiple knowledge users (patients, parents, healthcare providers, and researchers) have informed the design of both the exergame intervention and the trial. This registry-based trial will also identify the best tools to measure meaningful change in this population and provide the framework for future pragmatic trials in SMA.
CIHR recently featured Dr. Oskoui’s innovative trial in their “Faces of Health Research” initiative. Read the full feature to learn more!
Links
1. https://cihr-irsc.gc.ca/e/53357.html?filter=Maryam+Oskoui
2. https://www.informrare.ca/
Read next...
Defeat Duchenne Canada launches 2023 Research Grant Program
DDC invites research proposals for their 2023 research grant program of a translational or clinical nature focused on improving the health and quality of life for individuals living with Duchenne muscular dystrophy.
Early Career member Dr. Jean-Philippe Leduc-Gaudet identifies gene involved in regulating autophagy and skeletal muscle integrity
NMD4C early career member, Dr. Jean-Philippe Leduc-Gaudet, has made a significant discovery by identifying a novel gene that plays a key role in regulating autophagy and skeletal muscle health
New CIHR-funded Burden of Inherited Neuromuscular Disease (BIND) Study: Assessing the Indirect Socio-Economic Burden of Inherited Neuromuscular Diseases
The BIND study’s goal is to assess the social and economic burden of Canadians living with neuromuscular disorders by using web-based surveys to assess quality-of-life, healthcare resource use, work productivity, and effect on schooling and careers.
Muscular Dystrophy Canada, Canadian Neuromuscular Disease Registry, NMD4C partner to ensure Canadians can access Facioscapulohumeral muscular dystrophy (FSHD) cure(s)
MDC and the CNDR are working together with NMD4C to introduce a patient registry and collect information to build a Canada-wide core dataset for FSHD.
NMD4C Announces 2023 Early Career Award Recipients
We are excited to announce the recipients of the inaugural NMD4C Early Career Investigator Awards for 2023! This annual award program introduces three new awards, celebrating excellence and contribution to the neuromuscular field by early career investigators across both clinical and basic science streams.
