Organized by EURORDIS, Rare Disease Day has grown into a worldwide phenomenon over the last several years. We have continued our annual tradition of helping to spread awareness on Facebook with custom "I Love Someone Rare" profile frames. And you can share the love year-round with our exclusive MTM-CNM merchandise at our Zazzle store!

More info from RareDiseaseDay.org

Rare Disease Week on Capitol Hill brings rare disease community members from across the country together to be educated on federal legislative issues, meet other advocates, and share their unique stories with legislators. Our MTM/CNM community is well represented this year by MTM-CNM Family Connection's Vice-President Marie Wood and the Lykens family - as well as Myo the Traveling Turtle !

More info on Rare Disease Week

We are thrilled to have received a 2019 RARE Patient Impact Grant from Global Genes! This funding will go directly to supporting travel costs for families who might otherwise be unable to attend our conference in St. Louis this July. Stay tuned for more details and application process.

More info on Global Genes Rare Grants

The American Society of Gene & Cell Therapy (ASGCT) has developed some excellent educational materials on Gene Therapy in general, as well as XLMTM-specific information. We collaborated with ASGCT in the development of these materials and are glad to see they are being well received by the community!

More info from ASGCT

Creating a new treatment for a rare disease like MTM/CNM certainly has many technical, scientific and clinical challenges to ensure it is safe and effective. But even if a treatment is approved someday - will it be accessible and affordable? This is another important area of advocacy for our community.

Read about the PAVE Act here.