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2023 CMD Research Roundtable Launches Jan 28

 

A 10-month program for stakeholder community members to share and learn about science, current and potential future research, and to agree on attainable, realistic treatment goals based on priorities for symptom management. This program will provide participants the opportunity to become an active voice in directing the research roadmap for the five primary subtypes of congenital muscular dystrophy.

Aims

  • For affected community members to gain a realistic understanding of what it will take to get to clinical trials, new opportunities in scientific advancement, roadblocks to solve in taking those opportunities from theory to practical application, what treatments in the next 5-10 years are likely to be possible, and to be an instrumental (and informed) voice in determining CMD research priorities.

  • For researchers to have the opportunity to connect with the community they are doing science for, ensure that the work they are doing is responsive to community priorities, and to consider new approaches to treatments that extend and enhance quality of life on the road to identifying a "cure".

  • For other stakeholders, like industry, clinicians, policy makers, grant funders, regulatory agents, to gain a better understanding of CMD, the research landscape, treatment pipeline, and the opportunities on the horizon to translate bench discoveries to practical treatments.

  • For all participants: an opportunity for candid sharing and discussion about what is and is not likely possible in terms of treatments with our current knowledge and capabilities.

Affected Community Participation Requirements

  • Is an affected individual or parent/guardian of affected person with COL6, LAMA2, α-Dystroglycanopathy, LMNA, or SELENON (diagnosed at minimum clinically - prefer genetic confirmation)

  • Affected individual(s) is fully registered in the CMDIR and, if available, a genetic report is submitted for curation

  • Commits to attending at least 4 of 6 live virtual meetings, and for meetings missed; commit to watching the recording prior to the next meeting

  • Commits to registering and actively participating in discussions at least twice per month on the CourseNetworking platform (login information will be provided after registration)

  • Commits to completing all reading and watching assignments, and surveys/quizzes/polls on the CourseNetworking platform

Curriculum Overview

1st Meeting: All Subtypes
Review aims of Series, expectations for participation, vocabulary list, full series reading assignments with due dates, platform overview. Assigned survey/quiz following first meeting.

2nd Meeting: Subtype Specific
Basic overview of subtype pathology, therapeutic toolbox, current research underway, assets and challenges. Assigned pre-reading. Assigned survey/quiz. Share results of initial survey/quiz.

3rd Meeting: Subtype Specific
Basic Research: Up to 3 researchers pre-record a 5-7 min video about their current project: aims, assets, challenges, translational path. Assigned pre-reading. Assigned survey/quiz. Meeting is a 1-hour discussion/share results of recent surveys/polls, and Q&A.

4th Meeting: Subtype Specific
Translational Research: Up to 3 researchers pre-record a 5-7 min video about their current project: aims, assets, challenges, translational path. Assigned pre-reading. Assigned survey/quiz. Meeting is 1 hour discussion/results of survey, Q&A.

5th Meeting: Subtype Specific
Treatment vs Cure vs Care Optimization/QoL Tools: Discussion. Share Affected Community Priorities (surveys/discussion/Q&A). Challenges, likely outcomes for each subtype. Assigned survey/quiz. Meeting is 1 hour discussion/results of survey, Q&A.

6th Meeting: All Subtypes
Overview of series, lessons learned from researchers and affected community, final survey results/comparison to intiial survey, Cure CMD's future goals based on outcomes of project: Paper, Research Grant Priorities, New CMDIR Surveys, New educational content.

Online Interface

Cure CMD will use the CourseNetworking Pathway Learning Management System and Zoom to provide participants an online interface to meet, review assignments, complete surveys, polls and quizzes, and interact with other participants between live meetings. After completing registration, you will receive an invitation to create a free account with CN. You will also need to create a Zoom account if you don't already have one (also free). Please be sure the email you list below is one that you have access to and check regularly.

As mentioned above, participants are required to fully registered in the
Congenital Muscle Disease International Registry prior to the first meeting.
If already registered, we ask that you log into your CMDIR profile to ensure your contact and diagnosis information is correct, that all surveys have been completed, and a copy of your genetic report has been submitted for curation (if you have received genetic confirmation).  If you have already registered but do not remember your login information, please do not register again! Click the "forgot password link at the top of the page.

Registration Fee

We have elected to charge a nominal fee of $25 for this series to help ensure your commitment to full participation. This series only works if everyone registered is engaged throughout the series. 

There are no refunds for any reason. If you ultimately decide you cannot fully participate (prior to Jan 28, 2023), your payment may be converted to a donation and a contribution acknowledgement provided. Please note that donations are only acknowledged as such if no goods or services are exchanged, therefore, your fee cannot be converted to a donation after the series begins.

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