💜Wishing you COMFORT in knowing you are not alone, as we continue to work and advocate for all of our loved ones. 💜Wishing you JOY in the little things, valuing the gifts that our loved ones with MTM & CNM have given us. 💜Wishing you Health, Happiness, and HOPE in 2021!

Proud of our team member #StrongWill for sharing his story with the #FDA for #RareDiseaseDay #RDD2021 #mtmwarrior #PatientAdvocate #mtmcnmfamily #CareAboutRare #MyotubularMyopathy #RareDiseaseStories

Register to attend our Community Conversations together with WillCure #PatientEngagement #DrugDevelopment #myotubularmyopathy #willcuremtm #mtmcnmfamily #accesstotreatment4all

SAVE THE DATE for an externally-led Patient Focused Drug Development meeting with the FDA... conta.cc/3fDn45M

Register now for the Aug 3rd Externally-Led PFDD for the MTM-CNM community at mtm-cnm.org MTM-CNM Connection WillCure #RareAdvocacy #DrugDevelopment #PatientEngagement #accesstotreatment4all #myotubular #centronuclear U.S. FDA FDA Biologics FDA Patient Affairs FDA_ORA (Office of Regulatory Affairs) FDA CDER

Happening tomorrow-Aug 3rd! Come hear incredible individual & family stories helping to put the patient voice front & center in drug & therapy development!

Keep connected! Join us for an update with Dynacure this coming Monday about their clinical trial for the treatment of MTM & CNM! conta.cc/3FYpK9p

Join U.S. FDA #RegenMedEd virtual webinar on Oct. 27 to learn how to organize, execute, and participate in #NaturalHistoryStudies. Hear first-hand experiences from researchers, patients, and advocates. Free & open to the public. #CBER #OTAT Register: bit.ly/3DYdAyA

Very proud of Erin Ward Erin Ward & President of MTM-CNM Connection Thank you for being such a strong voice for our #myotubular and #centronuclear community #TNMD2022

Excited for MTM-CNM Family Connection to be participating in TREAT-NMD 7th International Conference, bringing together researchers, medical professionals, industry, advocacy leaders, patients & families to accelerate therapies neuromuscular conditions. #tnmd2022 #mtmcnmfamily

Please join us for this upcoming webinar: us02web.zoom.us/webinar/regist… #mtmcnmfamily

On February 28th, we honor and celebrate all of those who are impacted by rare diseases, especially loved ones in our #MTMCNMFamily. Join the global movement and spread awareness of #RareDiseaseDay.

Webinar: save the date! 📷 3pm UK 22nd April All invited to join a global panel of clinicians, researchers, and patient organisations to discuss how liver issues in MTM & CNM may impact patient care, as well as clinical trials and potential therapeutics: bit.ly/3GlckGa

Important webinar for the #mtm and #cnm #raredisease community! Come hear about initiatives to better understand liver issues and how you can help accelerate research. April 22nd 10 am ET!

Introducing the presenters for the first session of next week's webinar on Liver Issues in MTM & CNM. Register here us02web.zoom.us/webinar/regist…

Learn about the MTM-CNM Liver Collaborative Working Group & new initiatives to accelerate understanding of liver issues! "Liver Issues in MTM & CNM" this Saturday, April 22nd Co-hosted by @mtmcnmfamily Myotubular Trust & MTM & CNM Patient Registry us02web.zoom.us/webinar/regist…

Part 2 of webinar tomorrow will focus on "Understanding Liver Issues in the context of clinical trials". Come hear about info and learnings from the Aspiro #genetherapy trial. Astellas Gene Therapies Myotubular Trust @MTM & #CNM #PatientRegistry us02web.zoom.us/webinar/regist…

NYU Langone Health Division of Medical Ethics' working group on Pediatric Gene Therapy and Medical Ethics(PGTME) is recruiting for a study about the caregiver experience with pediatric gene therapy. #patientexperience #genetherapy #livedexperience

Liver information to share with your care team #mtmcnmfamily #newsletter myemail.constantcontact.com/Liver-informat…

Join the FDA Biologics's fall #RegenMedEd webinar on Oct. 5 at 11 am ET. Hear inspiring stories of advocacy from families of individuals with rare disease. Register now: bit.ly/3Rj5siN