Myotonic Dystrophy Foundation

Our Vision
We envision a world with treatments and a cure for myotonic dystrophy.

Our Mission
The mission of the Myotonic Dystrophy Foundation is Community, Care, and a Cure.

We support and connect the myotonic dystrophy community.
We provide resources and advocate for care.
We accelerate research toward treatments and a cure.
The Myotonic Dystrophy Foundation (MDF) is the leading global advocate helping patients and families navigate the myotonic dystrophy (DM) disease process, and is often the first resource contacted by newly-diagnosed patients, their families, their social workers and their physicians around the world. For many international patients, the Myotonic Dystrophy Foundation is often the only resource they are able to locate, and MDF has provided assistance and support for people living with DM in more than 139 countries around the world.

Currently tens of thousands of people living with myotonic dystrophy, their families & friends make up the Myotonic Dystrophy Foundation community.

Support & Education: Myotonic Dystrophy Foundation provides emotional support, education & resources to affected individuals & their families, & conducts outreach to raise awareness about DM worldwide. Resources & programs include: Toolkits and publications; phone support; comprehensive web resources; support groups & conferences.

Research: Myotonic Dystrophy Foundation is committed to advancing research & the search for therapies. Efforts include: Pre and Post-doctoral fellowships to expand the research base; managing the Myotonic Dystrophy Family Registry; Investing in target identification & drug screening; Supporting a clinical research network; determining the true prevalence of DM & documenting the socio-economic burden of the disease.

Advocacy: Myotonic Dystrophy Foundation advocates for legislation, research, & infrastructure initiatives that will advance research, accelerate drug development, & improve diagnosis & care. We raise the visibility & understanding of myotonic dystrophy with Congress, federal & state agencies, medical professionals, & the media.

The Myotonic Dystrophy Foundation has assembled an expert group of exceptional people we call on each day to drive toward therapies & better quality of life for our community.

The Foundation's Board of Directors is comprised of leaders in science, finance, fundraising, advocacy & non-profit leadership. Their expertise helps develop our organizational strategy, assess our impact, & guide our pursuit of patient-relevant outcomes.

The Foundation's Scientific Advisory Committee is an international group of leading myotonic dystrophy specialists & researchers based in industry and academia. They help develop the research platform and strategy and provide guidance on research investment decisions.

The Foundation's staff, which includes a CEO, Program Director and other professional staff, leads the day-to-day operations of our organization & works with the board to set & execute our strategic agenda.

Myotonic Dystrophy Foundation serves more than 15,000 DM patients & family members worldwide every year. We have significantly improved care for patients & families by developing the first-ever international clinical care recommendations for DM1, DM2, congenital DM, cardiology & pulmonology. MDF worked with the U.S. Social Security Administration to improve access to disability benefits for people living with DM and had congenital DM classified under its Compassionate Allowance program.

Myotonic Dystrophy Foundation executed a $5 million, 3-year drug development acceleration effort to attract more industry investment, lower barriers to therapy development, & expand the amount of data available to drive discovery. We fund research fellows, clinical trial infrastructure projects & other key research & maintain one of the largest DM patient registries in the world. Myotonic Dystrophy Foundation successfully advocates for additional DM funding at several federal agencies & works with regulatory agencies to optimize the drug review & approval process.