MTM-CNM Family News!!
June | 2021
SAVE THE DATE!!!

Join the MTM-CNM community on August 3, 2021 for an externally-led Patient Focused Drug Development (PFDD) Meeting
co-hosted by MTM-CNM Family Connection and Will-Cure!!
Will Cure
What is an "Externally-led Patient Focused Drug Development (PFDD) Meeting"?

The FDA states, “Patient-focused drug development is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation. As experts in what it is like to live with their conditions, patients are uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation”. 

So, a PFDD meeting is a platform for the patient community to share the stories of how their lives are impacted by the disease and their hopes and priorities when it comes to any potential therapies.

A PFDD meeting is not specific to any drug and therapy but rather a moment to hear the patient voice on the disease impact and what meaningful improvement looks like from the patient and caregiver perspective.

("Externally-led" means that the meeting is being run by the patient community rather than being hosted by the FDA.)
Tips When Recording Your Videos!

Don’t worry about a perfect video, the FDA wants to hear from you, not be impressed by your production skills. A few simple tips can help, though! 

• Hold your phone horizontally when recording 

• Get close and fill the frame with the subject 

• Use a tripod or stand if possible to keep the phone steady 

• Have bright, even light (a big window works great!) coming from the front or side of the subject, but not from behind the subject 

• Speak slowly and clearly. If you are nervous, record a session “just for practice” - it might turn out great! 

• Make use of pauses in between different video topics to allow for editing.

• If you stumble or mis-speak, keep rolling. Take a couple of breaths and give it another shot. (We can edit and just keep the good stuff). 

• Upload videos to https://www.dropbox.com/request/Ztm3qHOhLdrZtTE4pNjK (you do not need a Dropbox account, and your submitted videos will be stored privately and securely)

• You can upload one longer video, or break it up into shorter clips, whichever is easiest for you. If possible, include your family name in the filename(s).

• Please read instructions carefully and submit videos in the following categories:

1) Greeting/Intro/Goodbye Video [ALL are invited to participate!]
2) "If I Had a Little More Strength" Video
3) Life Impact Video
4) Potential Treatment Video
5) Daily Care Videos
How You Can Help Make the PFDD Meeting a SUCCESS for ALL of us!


You can participate by MAKING VIDEOS which we will incorporate throughout the virtual PFDD meeting to ensure the voices and faces of as many community members as possible are included in this pivotal community event!

This is NOT the story of two or three families nor of any single individual, event, or organization. This is about building a comprehensive and inclusive picture of our MTM-CNM world that can inform and guide those in a powerful position to help us!

We invite EVERYONE to submit a simple, SHORT video with the following:
 
• 3-5 seconds of you/your family waving “hello” 

• (PAUSE 3 seconds) 

• “Hi, we are the (name) family from (city/town). (Name) lives with Myotubular Myopathy”, or “Hi, I am (name) from (city/town), and I live with Myotubular Myopathy”. 

• (PAUSE 3 seconds) 

• 3-5 seconds of you/your family waving goodbye and saying “thank you”


OTHER VIDEOS you can make that can help tell the full story of living with MTM & our hopes/dreams to the FDA would be the following: 

“IF A HAD A LITTLE MORE STRENGTH” VIDEO 

Record a brief statement or two in your own words about what a little more muscle strength could help you accomplish … for example: 

• If John had a little more strength it would mean he could swallow without choking
• If John had a little more strength it would mean he could sit longer and go to school longer...
• If John had a little more strength he could go from non-verbal to verbal... 
• Spend some time off the ventilator 
• Play more video games 
• Type on an ipad. 
• Etc. 


IMPACT VIDEO: What do you want the FDA to know about how MTM impacts you/your family?

• Make a video that conveys an important part of your life with MTM. What do you think people don’t realize about your everyday life? How is your life different than your friends or extended family? How does MTM impact your social life, school, work, family and relationships, finances, etc? What supports (doctors, nursing, equipment, treatment routines, etc) do you rely on?

• Don’t need to introduce yourself 

• 2-3 minutes total talking about how MTM impacts you/your family/your loved one 


POTENTIAL TREATMENT VIDEO: What would you want from a potential future treatment for MTM?

A video that describes in your own words what would be meaningful improvement.

Even if not a complete “cure”, how could life be made better with a little more muscle strength? Same idea as the brief statement video above, but you can expand beyond the one line requested above for approximately 2-3 minutes. 


DAILY CARE VIDEOS: What medical care, treatments, routines, equipment do you rely on in your daily life?

A video showing interventions that help keep you healthy as possible with MTM:

• Describe what is a tracheostomy  
• Show and describe your ventilator 
• Video how you suction 
• Images of driving wheelchairs 
• Clips using adaptive equipment (lifts, assistive technology, bath chairs, etc.)

• If you decide to record more than one care, equipment, intervention, please pause for a few moments between each topic. 

The pauses are necessary to facilitate editing.
WE'RE IN THIS TOGETHER!!!
THANK YOU IN ADVANCE FOR YOUR EFFORTS!!!

This is an amazing opportunity for your voice and family to be heard by the FDA. Reach out if you have any questions along the way!!


**Deadline for videos is July 9th to allow for editing/compiling in advance of the August 3rd Virtual PFDD.
MTM-CNM Family Connection | www.mtm-cnm.org