The Neuromuscular Disease Network for Canada

Thank you to everyone who attended the Annual NMD4C Meeting on September 7th in Ottawa! We were thrilled to welcome 84 network investigators, general members, patient partners and trainees to outline the network activities for the next five years and kick off our new grant period. 

The meeting program consisted of presentations from task and theme leads with an opportunity for discussion from the network investigators present, along with breakout sessions on the topics of pre-clinical research and the clinical trial network. An overview of the network activities as a part of the new grant is available here. 

Additionally, the NMD4C highlighted early-career members by projecting profiles from 27 early-career members in attendance and hosting a networking session chaired by our 2023 NMD4C early-career researcher of year award winners, Drs. Nicolas Dumont and Cam-Tu Nguyen. This session showcased brief presentations from the chairs and the 2022 and 2023 postdoctoral and clinical fellowship recipients on their neuromuscular journey and research interests.

NMD4C members gather for a group photo at the annual meeting.

Task and Theme Presentations at the NMD4C Annual Meeting

Preclinical Science (Pillar 1) 

Animal and Cell-Based Research

Drs. Natasha Chang and Kessen Patten outlined the pre-clinical science pillar tasks, including centralizing a list of NMD cell/animal-based research labs and traineeships, developing a Resource for online publishing of research protocols, and outlining the plan for an annual research trainee summer school, as well as identifying the specialized needs of basic researchers in bioinformatics analysis of RNA-seq and scRNA-seq, and training in outcome measures in preclinical models.

Biobanking

In the absence of the biobanking lead Dr. Jason Karamchandani, Dr. Kessen Patten presented updates on the in-progress development of the virtual catalogue for Canadian neuromuscular biobank samples, and the virtual consent module for reconsenting clinical samples, and discussed the development and needs for a biobank start-up guide for clinical academics to launch their own biobanking practice.

Clinical Research (Pillar 2) 

Clinical Trial Network

Drs. Craig Campbell and Kerri Schellenberg outlined the specific components that must be covered in establishing a clinical trial network, with consideration of membership, stakeholder input, site engagement, and the development of shared SOPs and templates. Other logistical considerations included the coordination, operationalization and monitoring of the clinical trial network, and the development of a NMD clinical trial training program for trial coordinators. 

Participant Recruitment and Evidence Collection

Dr. Victoria Hodgkinson outlined launching a new Canadian Neuromuscular Disease Registry (CNDR) disease dataset for FSHD and at minimum two further datasets, with OPMD and MG identified as potential options. Further CNDR objectives of piloting new modules for systemic complications of NMDs and integration of post-marketing requirements for SMA and DMD datasets, and the launch of a patient portal on the CNDR website were also presented. 

Outcome Measures and Natural History

Drs. Colleen O’Connell, Jodi Warman-Chardon, and Cynthia Gagnon presenters outlined plans to establish a Canadian NMD Outcome Measures working group, develop the infrastructure for a Canadian Master Observational Platform, launch training modules for NMD outcome measures administration, establish pediatric and adult Canadian sites to contribute standardized imaging data to MYO-Share and MyoApp, and produce patient-reported outcome measures in NMDs for the CNDR.

Clinical Practice Research and Education (Pillar 3)

Community of Practice (CoP)

Dr. Cynthia Gagnon and Marie-Hélène Bolduc focused on the initial work of conducting needs assessment related to communication, education, and networking for NMD healthcare professionals related to their clinical practice and outcome measures. Initially engaging with physiotherapists, occupational therapists and nurses, the task will build a stronger membership within this group, identify the most effective means of communication, and provide meetings and training in outcome measures to generate real-world data. The task leads also seek input on current projects that could benefit from a community of practice among allied healthcare professionals, and mechanisms to ensure active involvement from the entire NMD4C community. 

Training & Curriculum

Dr. Hernan Gonorazky presented on the potential pathway from Royal College AFC to a Royal College subspecialty in neuromuscular medicine, while in the short-term including all neuromuscular and EMG fellowships as AFC-approved fellowships. Strategies to collaborate with other training networks to continue building the resources available for neuromuscular fellows, and strategies for improving the teaching pedagogy within the weekly training series hosted by the network. The concept of an educational roadmap with tailored training resources for neuromuscular clinical specialists was also outlined. 

From Trials into Practice: Treatment Access, Health Economics and Leveraging Evidence

Drs. Homira Osman and Jodi Warman-Chardon presented on the task goals of measuring the cost-analysis summary of novel NMD therapies and assessing the social and economic burden of NMDs, leverage evidence to advocate for data-driven access to novel therapies while providing guidance on delivering real-world evidence on NMDs for CADTH and payers, and enabling research about access to new therapies across provinces and communicate results to regional stakeholders. The Burden of Inherited Neuromuscular Diseases (BIND) study is in progress, and mentioned as a pilot study that will provide the evidence to inform reimbursement decisions for treatments in NMDs.  

Themes

Early-Career

Dr. Homira Osman shared the overarching goals of ensuring that the next generation of NMD scientists and clinicians are engaged in all network activities, and in addition to the training activities described in each pillar, providing leadership and training opportunities to EC researchers (clinical or biomedical), including: chairing and presenting webinars, leading and participating in all network activities and creating online profiles via the NMD4C website and EC blogs, as well as reaffirming the commitment to providing annual fellowship funding and early-career recognition awards. 

EDII/IDEAS

Dr. Kerri Schellenberg highlighted the expanding the definition from Equity, Diversity, Inclusion and Indigineity to Inclusion, Diversity, Equity, Accessibility, and Social Justice (IDEAS). Specific goals of this theme were presented, including introducing IDEAS themes in the ongoing NMD4C/MDC webinar series and existing neuromuscualr fellowship training, providing education seminar for female carriers of SBMA to build awareness, knowledge and support, and seeking input on additional projects as led by our community of persons with lived experience. 

Expert Patient Capacity Building

Dr. Kathryn Selby presented on the ImPORTND training modules completed in the first phase of the network, and emphasized that the ongoing work will include providing support for researchers to include patient partners from research conception through all the stages of the project. Through collaboration with MDC, the theme will provide training, matching, and funding to enable these partnerships in research. 

KT & Impact assessment

Dr. Homira Osman shared the vision of integrating KT activities across all tasks. The KT theme will seek to engage healthcare providers and researchers, including the development of a Knowledge Engagement Impact Assessment Toolkit to provide researchers with tools and training for evaluating their engagement activities and reporting on their research impact (to capture collaboration, engagement, research outputs, policy influence, economic impact, contributions to patient and broader society).