A decade of connections: US MTM-CNM Family Conference 2019

This year the MTM-CNM Family Connection hosted their sixth biennial US MTM-CNM Family Conference. Below they write about the event. Photos courtesy of Levi Gershkowitz of Living in the Light.

Erin, Mark and Marie from the MTM CNM Family Connection at the MTM - CNM Family Conference 2019.

Erin, Mark and Marie from the MTM CNM Family Connection at the MTM – CNM Family Conference 2019.

We are so grateful that we had the opportunity to celebrate ‘A Decade of Connections’ while hosting the sixth biennial US MTM-CNM Family Conference in St Louis, MO from  19 – 21 July 2019. Approximately 175 people attended the conference gathering 48 families and over 30 professionals from our rare disease community, as well as so many families represented in spirit who were not able to travel.

Our conference theme ‘Through the Gateway: Expanding Possibilities’” echoed St Louis’ iconic arch and gave our community an opportunity to reflect on ‘gateway moments’ that attendees have experienced on this rare disease journey – whether living with MTM/CNM, caring for someone, or working tirelessly for our community and look with hope towards the expanding possibilities before us.

The Kalejaiye family.

The Kalejaiye family.

We were grateful to have several of our lead researchers and pharma industry professionals working on behalf of our community join us and share the very latest on MTM and CNM research and advancements in treatments and also engage in an interactive clinical care discussion along with patients and families.   A strength of our conference is also providing medical professionals an opportunity to hear our patient and family stories, experiences, and insights as well, and collaboratively learn together.

Clinical Care Panel Discussion at the MTM-CNM Family Conference 2019.

Clinical Care Panel Discussion at the MTM-CNM Family Conference 2019.

Presentations covered such topics as genetics, diagnosis, clinical care, breathing, carrier issues, and the development of three potential treatments.  The three potential treatments that were presented at the conference this year included: Gene Therapy sponsored by Audentes, ASO Knockdown of DMN2 sponsored by Dynacure and re-purposing an existing drug (Tamoxifen) lead by Dr Jim Dowling’s Lab at The Hospital for Sick Children, Toronto.

We are also appreciative of the collaborative patient/family and professional discussion forum that followed the formal presentations on Saturday afternoon.  This year’s focus was on exploring clinical care guidelines and the potential need to expand resources and research in this area to ensure that optimal care is given to our loved ones.  It was an exceptional opportunity for both families and professionals to exchange information and experiences with each other in thoughtful and open dialog.

Attendees at the MTM_CNM Family Conference 2019.

Attendees at the MTM_CNM Family Conference 2019.

Our conference certainly highlighted the ‘Expanding Possibilities’ for our MTM-CNM community both in terms of promising treatments in development, as well as activating our community to become engaged partners and participants in the drug development process.  Our MTM-CNM Family Connection team held a panel on the importance of legislative advocacy and announced the development of COLA (Committee on Legislative Advocacy).  We will continue to educate, advocate, and activate our community to be the best advocates for our loved ones as we continue to pursue therapeutic treatments for all.

Families and individuals also shared valuable information about living with MTM and CNM and helpful resources with each other.  Strong connections were made over the weekend and the strength and support of our MTM-CNM Family Conference community was palpable. It gave us energy to continue our journeys and we made lasting memories to carry home with us.

US MTM-CNM family conference group photo.

US MTM-CNM family conference group photo.

 

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