We did it!
2013 MTM-CNM FC photo by Sergio
Photo courtesy of Sergio Vazquez
As you may know, the MTM-CNM Family Conference took place on July 26-28 in Bloomington, Minnesota! This is your post-conference update!

What Happened...
2013 conference Andy Welter
"It's not a conference. It's a party" ~Andy Welter

The theme of our conference was "The Road to Research, Resources, and Relationships!" In that spirit, we were delighted to greet MTM-CNM families from across the United States and Canada! Research teams from France, Washington, Wisconsin, Massachusetts, Michigan, and Florida also joined us to share the latest developments in their research!  In all, over 100 family members and 28 medical professionals were in attendance.

Family introductions, games, and raffle prizes kicked off the fun on Friday night! Some attendees also enjoyed free massages (or signed up to get one later during the weekend)! This was just a fun time of new people getting to know each other for the first time and for old friends to catch up with each other in person! 

Two promising lines of research for treatment of Myotubular Myopathy were some of the main topics for discussion. Dr. Anna Buj-Bello and Dr. Martin Childers presented the latest developments in gene replacement therapy, which is truly an exciting international collaboration. Dr. Mike Lawlor also presented exciting information about the developments in enzyme replacement therapy for MTM. Dr. Laurent Servais discussed some of the tools he has developed to measure muscle strength, which will help us evaluate the successfulness of various treatments.

Will Whiston Anna Buj-Bello and Woody
Will Whiston with Dr. Anna Buj-Bello...and Woody!

Dr. Dowling presented us with information about the Natural History study and how crucial his work is regarding the path to clinical trials. He and his team from the University of Michigan gathered data from patients on-site. Dr. Barry Byrne, Dr. Barbara Smith, and their team from the University of Florida also told us about their work in respiratory measures and the lessons they've learned from studying Pompe Disease and the implications it has for our community. The team from Florida was able to obtain a great deal of data from attendees with MTM and CNM through their observational study. Lindsay Swanson of the Beggs Lab at Boston Children's Hospital also met with families for genetic counseling, and Sabine de Chastonay connected with families about enrolling in MTM studies underway by the CMDIR.

While we have had researchers join us for conferences in years past, in 2013 we were thrilled to add a new dimension for our community: two new biotech companies that are dedicated to creating treatments for MTM were represented!  Matthew Patterson, CEO of Audentes Therapeutics, spoke about his plans to develop a treatment based on gene therapy.  Deborah Ramsdell, CEO, and Hal Landy, CMO, of Valerion Therapeutics shared with us their approach to developing a treatment based on enzyme replacement therapy.  Both of these have shown great promise in pre-clinical research, and between them, there is about $50M in financing committed to help accelerate the push to clinical trials!  It was wonderful to have them fully participating in the conference and spending time getting to know our families.

William and Louis
New friends, Louis Wilhelm and William Ward!

We learned about ways in which our families can get involved in contributing information for studies: the MTM/CNM Patient Registry (sponsored by the Myotubular Trust),  the Joshua Frase Foundation's global mapping project, the Congenital Muscle Disease International Registry and Event Study, the Congenital Muscle Disease Biobank, and the Congenital Muscle Disease Tissue Repository. 

Throughout the informational talks, we had fun activities for the younger children with a clown, balloon artist, face-painting, arts & crafts, impromptu little girl manicures, movies, sock-puppets, and more! During the afternoon breaks, families went out to the Mall of America for shopping, playing, relaxing, enjoying the rides of the Nickelodeon amusement park, and visiting the aquarium! The major highlight of fun took place on Saturday night with the variety show! After our parade of hats, we were entertained by singers, lip-singers, drummers, bloggers, readers, comedians, guitarists, and more! It was really great to see many of the families participate as well as to have the doctors, researchers, and therapeutic company representatives join in, too!

2013 Talent Show U of Washington
University of Washington team in the Variety Show

If you want to check out what other attendees are saying about the conference experience, read Hilary Sirmon's thoughts on Miracles to Milestones or Burke Stansbury's posts about it on his blog for Lucas.
You can also visit our conference Facebook page and our website, www.mtm-cnm.com, to see more of the pictures, lecture summaries, and videos as they are posted and updated.

2013 reflection room
Reflection Room
Who Made It Happen
Parade of Hats 2013
Parade of Hats!

Erin Ward (conference director), Mark Ward, Shannon Mashinchi, and I (Marie Wood) are truly humbled and gratified by the many kind words of thanks from people in our community for our contributions to this event and to this mission. While it is true that we have invested a great deal of our time and talents over the past two years of planning and fundraising, we also have many others to thank for helping us bring all the pieces together in the final product! 

We could not have pulled everything off without conference volunteers, like Sergio Vazquez (official conference photographer) and Patricia Ocampo (conference program designer), the Welters (our local family who is always available to help in any way possible), Krista Hanson (who coordinated the massage therapy sessions and led a yoga session) and Burke Stansbury (who along with Krista facilitated family panel discussions), Allie Welter and Debbie Majeau (our go-to Canadian, eh!) for organizing kids' activities, and Stesha Mashinchi for acting as a resource for teens and siblings of affected individuals. 

We thank the doctors and researchers, who committed their time and resources in order to share important updates and to gather data from our families and get to know our community better! We are also grateful for the financial support of our generous donors and for the Scoggin family, who allow us to raise tax-deductible donations for the conference through their non-profit organization, the MTMRG. 

There are so many essential elements, and we are grateful for all involved. Most especially, the conference is about building our community, if we didn't have families who were committed to getting to the conference, whether flying or driving (across the country, in some cases!), there would be no conference. Thank you so much to all of our conference attendees for the courage and sacrifices you made to be a part of this powerful event. You guys are awesome! We do this for you and for every one of the MTM-CNM individuals and families in "our world," who, even when circumstances do not allow us to be together in person, are forever and always with us in spirit!




What Will Happen Now
Erin and Marie
Erin Ward and Marie Wood
One of the most difficult parts of experiencing something so wonderful is having to leave and say "Goodbye" to all of our extended MTM-CNM family. Happily, however, the conference seems to have stimulated many in our community to continue strengthening these bonds! There is a lot of buzz about local family get-togethers and camping trips. How cool is that?! And just to let you know, our team is already pushing forward to what's coming up next for the MTM-CNM Family Conference! In order for us to serve the needs of this community, we need to know what you need. So get in touch with Erin, Mark, or Marie to share your suggestions and feedback about the conference! Keep an eye out for these updates and stay connected on Facebook, too! 
The 2013 MTM-CNM Family Conference may be over, but our mission continues!




The MTM-CNM Conference Planning Team!


MTM/CNM Family Conference
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