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Lead investigator

Collaborators & Co-Investigators

• Lena Xiao, MD (Co-PI)
• Kevan Mehta, MD (Co-PI)
• Deborah Olmstead, RRT
• Hanns Lochmüller, MD, PhD
• Homira Osman, PhD
• Kathryn Selby, MB, CHB, MRCP
• Nisha Cithiravel, RRT
• Nicola Worsfold, MSc
• Nouma Hammash, MSW, RSW
• Susi Vander Wyk
• Tori Lacey
• Victoria Hodgkinson, PhD

Research Sites & Affiliations

• The Hospital for Sick Children, Toronto, Ontario
• Alberta Health Services, Edmonton, Alberta
• BC Children’s Hospital Research Institute/University of British Columbia, Vancouver, British Columbia
• Canadian Neuromuscular Disease Registry (CNDR)/University of Calgary, Calgary, Alberta
• Children’s Hospital of Eastern Ontario (CHEO) Research Institute/University of Ottawa, Ottawa, Ontario

Budget: $50,000

Disorders: All Neuromuscular Disorders (cross-cutting)

Research Areas: Enhance Care ,   Amplify Research & Accelerate Knowledge

Abstract: 

Children with disorders that affect their muscles and nerves typically also have trouble with breathing. These disorders are called neuromuscular diseases (NMD). Some children with NMDs use machines to help with breathing and with coughing. These machines have been shown to improve quality of life and survival in these children, but may also be disruptive and hard to use. Most guidelines on the lung health of children with these disorders are based on what experts think because there is not enough research in this field. Recently, there have been new medicines for some of the NMDs. With all these changes to the care of children with NMD it is very important to prioritize the top research questions for scientists to focus on. This study will ask patients and families and clinicians about the questions they have about the lung health. The study has three phases. The first phase is a survey that collects research questions that are important. The second phase is a survey that narrows the research questions down to a short list. In the third phase, everybody comes together to decide on a list of the top ten research questions. This list will let people know about the most questions related to lung health for children with these neuromuscular disorders.

Impact: