MTM-CNM Family Connection
  • Home
  • About
    • About MTM/CNM
    • About Us
    • Contact
    • Newsletters
    • Traveling Turtle
  • Family Conference
    • 2019
    • Previous Conferences >
      • 2017
      • 2015
      • 2013
      • 2011
      • 2009
  • Resources
  • Research
    • Research Studies
    • Drug Approval Overview
    • Selected Publications
  • Fundraising
  • Blog


Our Blog

XLMTM Gene Therapy: Q3 2018 Update from Audentes

11/7/2018

0 Comments

 
PictureDr. Suyash Prasad, Audentes Chief Medical Officer, addressing the 2017 MTM-CNM Family Conference in Nashville
Yesterday, Audentes Therapeutics provided a quarterly company update, including information on their lead program - clinical development of AT132, a gene therapy product targeting X-Linked Myotubular Myopathy (XLMTM).  Their Phase 1/2 clinical trial, called "ASPIRO", is well underway, and they state:

"...we have recently completed enrollment of the Cohort 2 dose of 3x1014 vg/kg, including three treated patients and one untreated control patient.  We look forward to engaging with the FDA and EMA in the coming weeks to discuss development plans and the potential registration pathway for AT132, and to advance our goal of making this important treatment available to patients living with XLMTM as rapidly as possible."
​

Additional information in the full press release may be found on the Audentes website here.  Also, as has been their practice, Audentes has shared a letter with our community, which can be read here.
​


0 Comments

Considering Complex Spinal Surgery

10/29/2018

1 Comment

 
Picture
Erin Ward, President of MTM-CNM Family Connection, has been serving as a Parent Partner for the Boston Children’s Hospital process design team participating in an Agency for Healthcare Research and Quality-sponsored, Harvard Chan School of Public Health-led Engineering High Reliability Learning Lab, since the program’s inception in 2015.  The team’s work has focused on improving the process for complex spine surgery.  Today, the team's shared Decision-Making Guide and patient-centered video series has been released by the project’s collaborative partners at Courageous Parents Network.  Erin shares more in the blog post below:

Picture
In 2014, Will had major spinal surgery and although he rocked the surgery, the process to get there seemed so much more challenging and complex then it should be.  Much of the coordination to communicate perioperatively with Will’s multiple specialists was on us, his parents.  At the time I had been working with a complex care specialist on another project, and began to share the challenges we experienced.  He listened and heard the complexities of the process, and he believed that as a universal care system “we” could do better! Dr. Jay Berry was in the process of applying for a multi-institutional grant, which came to be known as “Engineering High Reliability Learning Labs" (EHRLL).  Under this grant, medical entities including our team from Boston Children’s Hospital would collaborate with Harvard’s Chan School of Public Health and Northeastern University’s Health Systems Engineering team to create better care processes using systems engineering principles, aiming to improve the quality of health delivery. 
 
Since 2015, our team has been working to improve the process that patients experience when considering, and undergoing, major spine surgery.  There are many learnings that have developed out of this work.  Today, the team's shared Decision-Making Guide, that we hope may be helpful for patients and families who are considering spinal surgery, was released by our collaborating partners at Courageous Parents Network (CPN).  CPN has also worked with our Boston Children’s Hospital team to develop a series of videos that share conversations about the challenges and considerations of complex spine surgery from the perspectives of a parent, complex care physician, and surgeon.  We hope that the conversations captured will allow patients and families facing a potential surgery an opportunity to consider how to go through this complex decision making process.  We encourage families to partner with their providers and work to develop a personalized care pathway that will be best for their child and family.  Please feel free to share with those that might benefit and also please share any feedback as the shared Decision-Making Guide is a living guide, intended to be updated and modified to best serve families and medical professionals partnering with them to provide optimal care. 

1 Comment

Welcome to Nashville, Y'all!

7/15/2017

2 Comments

 
As we approach our 2017 MTM-CNM Family Conference in Nashville, we are please to share this special guest post written by MTM Dad and Nashville resident Patrick Bowers.  Thank you Patrick for sharing this great information!

Five and a half years ago Molly (then 16) made her first trip to Nashville. She came to be part of a Nashville Song Writers International (NSAI) event. NSAI is a songwriters networking, education and lobbying group. Between education and first hand experience writing and performing in town, Molly had written a few songs and was encouraged by some accomplished musicians to explore songwriting further. Molly will tell you that from her first song, she knew it was what she wanted to do and she'll also say much of her inspiration comes from her brother Patrick and the ability he had to touch the lives of so many people. On that first trip and on every trip after that, Molly received more and more attention and was encouraged to relocate here if she wanted to pursue this as a career. Molly asked if we all would consider a move to Nashville and although there was some thought that went into it, the decision seemed to be an easy one for our family. I had retired from Law Enforcement. Carol, a Critical Care Nurse knew her skills could be utilized anywhere. Sophie, our 9 year old, (then 5) was getting ready to start Kindergarten and wanted to be near her sister.  However, Sophie holds onto her Connecticut roots refusing to use southern words like "Y’all." :) With that and Molly’s promise that she would get a college education, we moved to Nashville four years ago this month. Molly just graduated in May from Belmont University. 

          Although it might surprise many newcomers, Nashville also has a lot to offer besides cowboy boots and country music. It truly lives up to its two nicknames. "Music City, USA" might be self explanatory, but many don't know it's other nickname, "The Athens of the South." It even has a full-scale replica of the Parthenon located off of West End in Centennial Park! For families, the Adventure Science Center, the Nashville Zoo, the downtown walking bridge, and the cities parks are great places to visit. For the sports fan, our Preds happened to go to the Stanley Cup this year. How did your team do? We were surprised just how culturally diverse Nashville is with residents from all around the country and the world, in part due to its thriving economy. In fact there are estimates of 100 people a day moving to Nashville. Downtown Nashville has gained national attention with its nightlife and live music that begins at 10am and doesn't end until 4am. While Honky Tonk's, Boot Stores, Buskers, and yes, many Bachelorette Parties line both sides of lower Broadway, yet many Nashville neighborhoods offer so much more.

          With neighborhoods like Germantown, The Gulch, 12th Avenue South, Sylvain Park, and Edge Hill there’s a great walk-ability factor and the restaurant scene here rivals any major city in the country. East Nashville is perhaps our favorite with its artistic, musician, and hipster population. Farm to table restaurants like, “Husk” and “City House” live up to their name with local flavor and ingredients. BBQ joints, “Peg Leg Porker”, “Martin’s”, and “Edley’s” are finger licking good! For true Southern cooking, head to “Loveless Café” or “Monell’s”, and of course you can’t leave town without trying Nashville’s famous Hot Chicken. If you’re looking to spice things up, head to “Hattie B’s” or “Princes”.

          Nashville has become very progressive, but it has maintained its southern charm and historical significance. The Belle Mead Mansion offers a realistic view of plantation life in the 1800's, and in addition happens to be a winery. With over 700 churches, the largest number of any major city in the U.S., many Nashvillian’s claim it as the buckle of the Bible Belt. The Ryman Auditorium, known as the "Mother Church" of country music, is located just off of Broadway. Its acoustics are unmatched and it is one of the best venues in town. Nashville is also home of Music Row, which is now listed as a historical national landmark. Many songwriters and top musicians make their home here and in fact the recording facilities here, in every genre, are some of the best in the world. You might even see a star you know around town!
​
          Although our son Patrick had been gone a few years when we attended the first family conference in Houston, we traveled a great deal with Patrick and understand the effort that goes into making a trip like this happen. We’re extremely excited that MTM-CNM Family Connection chose Nashville as this year’s site for the conference and consider ourselves blessed to be a part of such an amazing community. We truly believe that the efforts within the MTM-CNM family can make a difference and we are grateful for all those who have done so much to fight this disease. Welcome to Nashville, Y’all!
 
The Bowers Family
 
(If we can assist with any questions, feel free to contact Patrick at pmbowers@sbcglobal.net)
Picture
You know about "Music City", but what else do you know about Nashville?
Picture
The Parthenon at Centennial Park
Picture
Nashville skyline from across the Cumberland River
Picture
Live music can be found just about any time
2 Comments

The Time Is Now!

3/27/2017

1 Comment

 
Picture
Hey y’all!  July is just around the corner, and we need your help!  Time is running out to get the needed funds raised for the 2017 MTM-CNM Family Conference in Nashville!

An unparalled experience
If you’ve been to one of our previous MTM-CNM Family Conferences, you know just how special they are.  If you haven’t been yet, here is what some past attendees have said:
  • “There are no words to say how special this weekend was to us. I feel like a part of our family is now complete because we finally were able to see in person part of our extended family. This weekend was so good for our hearts and soul.” - Natalie, Cooper's mom
  • “We found our Utopia this weekend in a Best Western Plus in Bloomington, Minnesota at an MTM-CNM Family Conference.” - Hilary, Benjamin's mom
  • "It's been the most valuable opportunity for me personally and as a scientist to be able to come and learn from the boys and their families." - Dr. Barbara Smith, University of Florida
  • “I've waited 19 years to feel this way and I will try to never miss this again. To feel the belonging and to talk to others who understand the day to day of my life and "get it" is just great!!!” - Christiann, Jason's mom
  • “You will never know how much this conference meant to me and my family. It was the most incredible, healing, convalescent experience I have ever taken part in. To be face-to-face with the researchers and have the space to ask questions and hear presentations, was so wonderful and incredible.  And of course our families! You gave us the opportunity to meet and share and laugh and cry!” - Maggie, Lincoln's mom
  • “We attended this conference two summers ago in Chicago Illinois and it was the best experience we ever had. Not only did we get to meet other families like ours our son got to meet other kids like him and feel normal for a change. We also got to listen to doctors and researchers who were studying our son's particular genetic disorder, so it was a great learning opportunity.” - Kimberly, Evan's mom
  • “It’s hard to describe how important being at the myotubular myopathy conference has been for us and all the other families here this weekend.  Sure, there was lots of fascinating and groundbreaking medical research presented to the group as part of the rather remarkable progress that has been made towards a treatment for x-linked MTM.  But most importantly for us, being at the conference was about connecting with, sharing, and learning from other families of kids born with MTM.” - Burke and Krista, Lucas's parents

The cost of the Conference
While the value to families and researchers alike is priceless, overall the conference weekend costs us nearly $300 per person.  The bulk of the cost of the conference comes from the hotel facilities, meeting space, catering and service, which translates to about $40 per person for each lunch and $60 per person for each dinner.  Other expenses include kids activities and supplies and various other related services that make the conference special.  This does not include the cost of hotel rooms which are paid by the attendees.

Who pays for it?
We are extremely grateful to be lining up a number of generous corporate sponsors again for the 2017 MTM-CNM Family Conference.  These are organizations who support our community and our efforts, and have some vested interest in helping to gather as many of us together in one place as possible.  Like us, they seek to drive research forward towards effective treatments, and to support families in every stage of dealing with MTM/CNM.  We couldn’t do this conference without them.

However, despite their generosity, sponsorships do not cover all the expenses of our conference.  After all, this is a family conference, created by families, run by families, and with families at the heart of the whole experience.  And we want all families to be able to participate, with as little direct cost to them as possible.  Therefore, we make this heartfelt appeal:

We need your help!
We are asking each family to help make this conference happen, by raising $500 or more for MTM-CNM Family Connection, by May 1st.  If you can raise even more, that would be wonderful.  If you can’t raise that much, we are grateful for whatever you can contribute - even if it is just words of support.  The more money that we can raise as a community - as a family - the better we will be able to serve each other and continue to make a meaningful difference in the lives of those affected by myotubular and centronuclear myopathy.  Let’s make April 2017 a month of amazing MTM-CNM fundraising!  May 1st isn’t a hard deadline - our fundraising goes year-round - but we need to have our financial picture clear as we get closer to the conference in July.

If you have the ability to make a $500 or more donation yourself, that is of course welcome, but what we are really hoping for is active participation in some form of fundraising.  This helps beyond just the dollars: it gets more people contributing, which means more people involved and aware and joining us in our mission.

How to get started?
The simplest way that anyone can get involved in this fundraising is through our partnership with Crowdrise.  Go to https://www.crowdrise.com/2017-mtm-cnm-family-conference and click “join the team” to set up your own profile page.  It just takes a couple minutes!  You can add your own photos and tell your own “story” if you wish, describing what the conference means to you.  Then share your page with family and friends to request donations - easy!  Just 20 donations of $25 each will get you to the $500 target.  The donations are tax-deductible and transactions are handled securely by Crowdrise and Network for Good.  If you need any help setting up your page, just ask!

There are a number of other ways to get involved with fundraising, from hosting online product sales (we’ve recently benefited from LuLaRoe, Pampered Chef, Thirty-One, Younique) to 5k runs, yard sales, lemonade stands, “jeans days”, race sponsorships, and whatever else you can think of!  If you want to do something but aren’t sure what (or how), feel free to contact us via email or start up a discussion with other families in our closed Facebook Group.

If fundraising falls short of our goals, then we will have to cover more of the costs through registration fees, which we would rather not do since higher costs may make it difficult for some families to attend.  So as an additional incentive, we will be offering $100 off registration for every $500 an individual or family raises.

Just do it!
The most important thing is to be involved - however you are able.  Families, this is our conference.  We can’t wait to see you there, and we thank you for helping to make it happen.  Let’s do this together!

1 Comment

Dynacure appoints its Management Team and members of the Board of Directors

3/9/2017

0 Comments

 
Picture
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!

0 Comments

Give Like It is Tuesday

11/29/2016

1 Comment

 
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.
As our community grows, and as research progresses down exciting yet uncertain paths, our mission is more important than ever, while the costs of putting on the Conference are increasing with this growth. With anticipated record numbers for the 2017 Conference in Nashville, the venue expenses in particular will be rising.  Please note that the money we raise goes 100% to our mission - only about 1% is for necessary overhead such as our website and email expenses, and the rest goes to the direct costs of the Conference weekend.  As always, our small team is all-volunteer and we never use funds raised for our own expenses, even when we are engaged in Conference-related work or travel.  And so, with deep gratitude we humbly ask for you to dig a little deeper on this Giving Tuesday and help support MTM-CNM Family Connection.
Picture
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!
Picture
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!

1 Comment

Top Tweets for October

11/1/2016

1 Comment

 
Here are the top tidbits and tweets from MTM-CNM Family Connection for the month.  Thanks for staying connected!

Congrats to team @willcuremtm raising $$ for research by running #ChicagoMarathon! Nothing can stop a determined #mtmcnmfamily!

— MTM-CNM Connection (@MTMCNMFamily) October 9, 2016

What exactly is Myotubular/Centronuclear Myopathy? Here are some smart people to tell you about it: https://t.co/nC3iF7ND4W

— MTM-CNM Connection (@MTMCNMFamily) October 3, 2016

Conference 2013 memory: Parade of Hats! Love these crazy kids. #mtmcnmfamily pic.twitter.com/Mz9rzYax7m

— MTM-CNM Connection (@MTMCNMFamily) October 19, 2016

Enjoying #worldseries? Root for athletes w/ MTM/CNM who play ball w/ @LLBChallenger! This & more resources at https://t.co/3HkFkXm105 pic.twitter.com/P7VqKqeymU

— MTM-CNM Connection (@MTMCNMFamily) October 27, 2016

The water can be rough. Keep paddling. #mtmcnmfamily pic.twitter.com/x1ityEVNVY

— MTM-CNM Connection (@MTMCNMFamily) October 28, 2016

Myo, we need you now more than ever! #voteformyo #travelingturtle #mtmcnmfamily https://t.co/e2p0CuWHwd pic.twitter.com/2xm9rxQpGX

— MTM-CNM Connection (@MTMCNMFamily) October 30, 2016

#genetherapy is not like other drugs. FDA's Janet Woodcock, MD discusses technical challenges w/@raredr https://t.co/mFIXxLZE3V pic.twitter.com/kSxKPpnHcL

— MTM-CNM Connection (@MTMCNMFamily) October 26, 2016

Updated Q&A on XLMTM #genetherapy development from Audentes, now available on our research page https://t.co/T3VbbRuvhr #INCEPTUS #ASPIRO

— MTM-CNM Connection (@MTMCNMFamily) October 13, 2016

Get smart! Check out our list of scientific papers about MTM/CNM, available to read online: https://t.co/73V2J30dgr #knowledgeispower

— MTM-CNM Connection (@MTMCNMFamily) October 7, 2016

Donations go to the #mtmcnmfamily Conference. We take no salary, minimal overhead, & pay our own personal expenses. https://t.co/xP8Ljw7MMa

— MTM-CNM Connection (@MTMCNMFamily) October 16, 2016

Check out our most recent newsletter about the 2017 MTM-CNM Family Conference! https://t.co/yFjY4QrMsc pic.twitter.com/zf44aPicES

— MTM-CNM Connection (@MTMCNMFamily) October 31, 2016

Today only! 25% off all MTM-CNM "Brave" merch w/code BLOWOUTSALE https://t.co/z8B3y6yzpD Support #mtmcnmfamily

— MTM-CNM Connection (@MTMCNMFamily) October 10, 2016

Who is excited to see their #mtmcnmfamily in Nashville July 2017? We are! https://t.co/Rr2u0Y5jnQ

— MTM-CNM Connection (@MTMCNMFamily) October 10, 2016

Connections matter. #mtmcnmfamily Donate at https://t.co/k2hFGDh6EW pic.twitter.com/ZmeeJ7grdV

— MTM-CNM Connection (@MTMCNMFamily) October 31, 2016

Tears of joy, tears of sadness. Such a privilege to be "on the road" with so many amazing people. https://t.co/FIT0PNve3f

— MTM-CNM Connection (@MTMCNMFamily) October 21, 2016

Resources for & from #mtmcnmfamily: https://t.co/UD1OnC3kd3 Let us know if you have a resource to share!

— MTM-CNM Connection (@MTMCNMFamily) October 17, 2016

Wow, what a bowl of spaghetti! And just one little point mutation somewhere in there can be responsible for #RareDisease like MTM/CNM. https://t.co/o7vqeMGNah

— MTM-CNM Connection (@MTMCNMFamily) October 31, 2016

What an easy way to support a great cause! Help make our #mtmcnmfamily conference a reality just by shopping! https://t.co/6gUTWxgEfn

— MTM-CNM Connection (@MTMCNMFamily) October 12, 2016

2017 @MDA_Advocacy Conference, Apr 23-25 in D.C., great opportunity to make the voices of #mtmcnmfamily heard! https://t.co/XdLXoITxWH

— MTM-CNM Connection (@MTMCNMFamily) October 22, 2016

NY-area families, check this out: https://t.co/p5cfGyioak https://t.co/Nw9dUpxQBj

— MTM-CNM Connection (@MTMCNMFamily) October 4, 2016
1 Comment

2016 Global Genes RARE Patient Advocacy Summit

10/15/2016

1 Comment

 
Picture
PictureMarie, Erin, Shannon, and Myo representing MTM-CNM Family Connection at Global Genes 2016 RARE Patient Advocacy Summit in Huntington Beach, CA
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!

Picture
MTM Families with Barbara Wuebbels, VP of Patient Advocacy Audentes Therapeutics. Left to right: Daniel McDermott, Erin Ward, Barbara Wuebbels, Christi Rocha, Marie Wood, Diana Manny, and Shannon Mashinchi
At such an important time for our community, at the threshold of clinical trials, we encourage all family members to learn everything we collectively can about moving into clinical trials, facilitating the liberation of medical data for researchers, collaborating with pharma industry to streamline the process of drug approval, and all becoming patient advocates for what is best for our loved ones.  We were thrilled other family members from our community also attended the summit and together we were empowered with valuable information and resources to support our community’s journey.  We also had an opportunity to connect with Barbara Wuebbels, VP of Patient Advocacy Audentes Therapeutics, and a leader in patient advocacy in the pharma industry.  For those that were unable to attend but would like to learn more, please visit the Global Genes website to view videos of the summit presentations and also check out the other valuable resources available for all rare communities.
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.
A month prior to heading out to California, we posted our  plans to attend this wonderful summit on Facebook with an open invitation to meet with any families in the nearby vicinity who were available. Two of our local families--parents of two MTM angels--came to visit us at our hotel for an all-too-brief but deeply treasured time of sharing, reminiscing, and encouraging. There were also lots of baby cuddles, which was the sweetest part of all! We were so happy to have a chance to connect while we were in the area.  There is nothing like spending time with MTM & CNM Family! 
     
Our team's experience at the 2016 Global Genes Patient Advocacy Summit was outstanding and we were grateful for the opportunity to participate in this RARE advocacy conference!  
Picture
Local MTM families, including Nancy and Maya Serafano (left) and Renee and Randy Lyde (right) were able to stop by for a visit with our team
Picture
1 Comment
Forward>>

    MTM-CNM Family Connection's Blog

    Sharing information, resources, updates, and thoughts on various topics of interest to the MTM-CNM community.

    Archives

    July 2020
    November 2019
    October 2019
    July 2019
    June 2019
    May 2019
    February 2019
    January 2019
    November 2018
    October 2018
    July 2017
    March 2017
    November 2016
    October 2016

    Categories

    All
    Patient Advocacy
    Patient Care
    RARE Disease Community
    Research News

    RSS Feed

Picture
Copyright 2014-2022 MTM-CNM Family Connection, Inc 
Admin
  • Home
  • About
    • About MTM/CNM
    • About Us
    • Contact
    • Newsletters
    • Traveling Turtle
  • Family Conference
    • 2019
    • Previous Conferences >
      • 2017
      • 2015
      • 2013
      • 2011
      • 2009
  • Resources
  • Research
    • Research Studies
    • Drug Approval Overview
    • Selected Publications
  • Fundraising
  • Blog