We are pleased to share this news from Dynacure, a new biotech company developing a novel treatment approach for Centronuclear Myopathies: http://www.dynacure.fr/news/dynacure-appoints-its-management-team-and-members-of-the-board-of-directors/
At our 2015 MTM-CNM Family Conference in Chicago, Dr. Johann Bohm from IGBMC in Strasbourg presented some of the early scientific work from Dr. Jocelyn Laporte’s lab (supported by a research grant from the Myotubular Trust) which has led to the creation of Dynacure in 2016. Dr. Belinda Cowling also presented on this topic at the 2016 European Family Conference in Germany (organized by ZNM-Zusammen Stark and Myotubular Trust). For a short summary of the theory behind Dynacure, please check out the article by Dr. Cowling on page 3 of this newsletter from ZNM-Zusammen Stark.
We are grateful for Dynacure joining as a sponsor of our 2017 MTM-CNM Family Conference, and we look forward to hearing more from them this July in Nashville!
As the year is coming to a close and the holiday season upon us, our hearts are warmed by the thoughts of our amazing community of family and friends who make our “MTM-CNM Family” so special. We are thankful for you in so many ways. In particular, as today is “Giving Tuesday”, we want to thank all of those who support our mission financially. Giving Tuesday is a wonderful tradition formed in response to the commercialism of Black Friday and Cyber Monday, and shifts the focus from “buying more stuff” to supporting important causes that make the world a better place. The MTM-CNM Family Conference literally would not exist without the support of families and sponsors, and we have heard from families and medical professionals following each Conference, of the life-enhancing and life-changing experiences that happen in our community because of the connections that are created and sustained through the Family Conference.
We are excited to offer a new way to support our efforts, that we hope many will utilize. We understand that it is not always easy to make a large donation - sometimes the money just isn’t there and bills have to be paid. And so we have created a budget-friendly tool: automatic recurring donations. If you had the opportunity, would you be willing to buy someone in your MTM/CNM family a drink from Starbucks once a week? Or perhaps get together for a pizza or dinner out once a month? If only we were all closer together! So do the next best thing - invest that money in community connections made through the Conference! When you set up a recurring donation, you choose the amount and the frequency and your contributions will be made automatically - it is super easy! Perhaps you can commit to $5 a week, or maybe $50 a month - whatever the amount, you will be making an ongoing commitment to MTM-CNM Family Connection and helping make the Conference a wonderful experience for all. Donations are tax-deductible and can be cancelled at any time. Get started today!
We’re also looking to expand our fundraising team at Crowdrise! This is a fun and easy way to partner with us and help us meet our financial goals, with our primary goal to keep expenses as low as possible for families to attend the Conference. By joining the team, you can set up your own profile where you tell your “story” of what the Family Conference means to you or your connection to our community. Then be as creative as you like in spreading the word and gathering donations from your family, friends, coworkers, etc. Donations can be made directly through your Crowdrise page or collected offline and credited to you (for example, if you want to hold a fundraising event). All the tools are there for you to track your progress, thank your donors, update your followers, etc. There is no minimum amount required to raise, but we encourage you to dream big! If you want to help but have questions or aren’t sure how to get started just contact us.
However you are able to give - whether financially, or by sharing your time and talents, or even just words of encouragement and support - we are grateful. We couldn’t do it without you. Thank you for being our #mtmcnmfamily. Hope to see you in Nashville next July!
Here are the top tidbits and tweets from MTM-CNM Family Connection for the month. Thanks for staying connected!
Members of our MTM-CNM Family Connection Team recently attended the 2016 RARE Patient Advocacy Summit, hosted by Global Genes, September 20th & 21st in Huntington Beach, CA. With the beautiful backdrop of palm trees and the Pacific Ocean and in an atmosphere of innovation, energy, and alliance, patient advocates from the greater RARE disease community came together to learn from each other and from the scientists, researchers, philanthropists, artists, and others working hard to support and spread awareness on behalf of our communities.
One of the primary takeaways from this conference was that although each individual rare disease community is small, there are over 7,000 identified rare diseases with millions being affected. That means almost everyone, whether aware of it or not, knows someone RARE. Sadly, only 5% of these diseases currently have an FDA-approved treatment. However, this is where the cumulative power of RARE can make a difference, and Global Genes has really stepped up to the plate in providing support and learning opportunities for everyone at every stage by leveraging the strengths of each community's advocates.
During breakout sessions, RARE advocates who had succeeded in particular areas, such as community building, establishing advisory boards, fundraising, soliciting volunteers, utilizing social media to spread awareness, coordinating longitudinal studies, and pushing forward on the path of clinical trials, eagerly shared what had worked and what had not, often opening up the floor for feedback and participation. The roles of teacher and learner were fluid and dynamic as advocates spoke to their strengths and then sought mentorship in areas needing to grow. So speakers in one round of breakouts would be audience participants in the next round.
For us, it was valuable to glean knowledge and insights from other communities that are further along in clinical trials, and we were happy to share our expertise with other rare disease groups that are just getting off the ground or planning their first family conference. The conference presentations were informational, the spirit of collaboration palpable, and the universal commitment to work until all rare diseases are curable, undeniable! Galvanized by this positive, exhilarating experience, we came away more committed than ever to continue in our mission to connect MTM & CNM families to essential resources, active research, and life-enhancing relationships!
Myo, our MTM-CNM Traveling Turtle, also made the trip and made his rounds at the summit, meeting patients, family members, researchers, pharma industry and government agency leaders. We had an opportunity to share with other patient groups how Myo travels around the world with the goal of connecting our community and spreading awareness of MTM & CNM. Myo was happy to meet some new friends and also did some advocacy work as well, supporting the #CURESNOW campaign through social media.