Dr. Hanns Lochmüller, NMD4C and MDC named co-investigators on two CIHR grants awarded for research into oculopharyngeal muscular dystrophy – led by Dr C Gagnon – and into the Indirect Socio-Economic Burden of Inherited Neuromuscular Diseases – led by Dr J Warman

The Lochmüller Lab is happy to share that project applications ‘A comprehensive study of the natural history of OPMD: An essential step towards clinical trial readiness and evidence-based interventions’ and ‘BIND Study: Assessing the Indirect Socio-Economic Burden of Inherited Neuromuscular Diseases’ have been awarded Canadian Institute of Health Research (CIHR) research grants in their recent round of funding decisions!

 

A comprehensive study of the natural history of OPMD: An essential step towards clinical trial readiness and evidence-based interventions

This project has been awarded $820, 846 over a 5-year period, and is led by investigator Dr. Cynthia Gagnon, Professor at the University of Sherbrooke and visiting professor at uOttawa and CHEO-RI, with Dr. Hanns Lochmüller as a co-investigator. Other investigators and applicants on the project include Dr. Bernard Brais, Dr. Élise Duchesne, Dr. Luc Hébert, Dr. Benjamin Gallais, Dr. Homira Osman, Claudia Côté, Dr. Xavier Rodrigue, Dr. Djamal Barbiche, Dr. Nancy Presse, and Dr. Jean-Denis Brisson.

The study involves four data collection sites in Saguenay, Québec, Montréal and Ottawa and will address the impact of OPMD over time in the lives of those affected by these neuromuscular disorders.

In addition to dysphagia, the team will document several characteristics related to physical performances, such as walking capacities and muscle strength. The study will also look at participation in daily activities, such as the ability to move in house and in communities, and to perform sports and leisure activities. The research team will also be able to select the best instruments to assess the key problems in this disorder such as muscle weakness and fatigue.

This study will provide essential data to help speed up therapeutic trials in this population by documenting natural history and informing outcome measures selection.

If you would like to learn more about this project, the scientific summary can be found here!

 

BIND Study: Assessing the Indirect Socio-Economic Burden of Inherited Neuromuscular Diseases

This project has been awarded $449,030 over a 3-year period, and is led by Dr. Jodi Warman Chardon, Director of the Ottawa Neuromuscular Centre in The Ottawa Hospital, with Dr. Hanns Lochmüller as an investigator. Other investigators on this project include Dr. Lawrence Korngut, Stacey Lintern (MDC), Dr. Kednapa Thavornas, Dr. Cynthia Gagnon, Dr. Hugh McMillan, Dr. Kathryn Selby, Dr. Homira Osman, Dr. Daria Wojtal, and Dr. Gerald Pfeffer.

The project’s goal is to assess the social and economic burden of Canadians living with genetic neuromuscular diseases (NMDs) by using web-based surveys to assess quality-of-life, healthcare resource use, work productivity, and effect on schooling and careers. The project team consists of Canadian experts in genetic NMDs, health economics and importantly, patient partners. The project consists of two major aims:

 

Aim 1: Estimate social and economic impact focused on the indirect costs and social burden of patients living with inherited diseases and caregivers. Individuals with genetic NMD and their caregivers will be invited through the National Muscular Dystrophy Canada advocacy group to complete a cross-sectional survey that aims to measure patient characteristics, treatment, disease burden, direct and indirect healthcare costs, and impact on work and school. These indirect costs will also be compared with those associated with common diseases (including Diabetes, COPD).

Aim 2: Determine the relationship between disease management and the social and economic burden of genetic NMDs. The group will use statistical analysis to assess how patient characteristics and disease management are associated with health-related quality of life and economic costs of genetic NMD. By assembling one of the largest cohorts of patients with genetic disorders in Canada, the results of this study will allow patient organizations and government to make informed decisions, influencing policy in health and health system planning to better support patients with genetic NMDs.

 

The grant summary can be found on the CIHR funding decisions page here.

 

 

The Lochmüller Lab is excited to contribute to these projects and collaborate with Muscular Dystrophy Canada (MDC), the Canadian network for neuromuscular diseases (NMD4C) and other members of the wider research community across Canada.

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