MDF Advocates Unite to Increase DM Research Funding

Published on Wed, 05/25/2022

MDF Advocates from Across America Urge Congress To Increase Research Funding to Cure Myotonic Dystrophy

Myotonic Dystrophy Foundation (MDF) advocates from across America participated in our first Advocacy Week campaign the first week in May to urge our Representatives and Senators in Congress to increase federal funding for myotonic dystrophy (DM) research to help us find a cure. The campaign was led by the MDF’s new National Advocacy Committee (NAC) and included a suggested action each day of the week. 

Rebecca Coplin from Oregon, a MDF NAC member and hospital executive, whose family is living with DM said, “Myotonic dystrophy remains one of the least funded rare genetic disorders.  Advocacy Week is an important part of our work to get Congress’ attention so we can increase federal research funding to find treatments and a cure.  They need to hear about our experiences to understand how important this is – it makes a difference. ” 

A Week of Action!

The week started on Monday, May 2nd with advocates invited to prepare for their virtual meetings with their Senators, Representatives, and their staff by reviewing the MDF Advocacy Week webinar that provided instructions on how to request meetings and send emails to Congress in support of our research funding requests. Advocates who visited were able learn about our campaign to obtain, for the 6th year in a row, DM research funding through the Department of Defense Peer Reviewed Medical Research Program (PRMRP), secure new DM research funding as part of the Congressionally Directed Medical Research Program (CDMRP), and initiate a new National Institutes of Health (NIH) research effort focused on DM and related repeat expansion diseases. 

On Day Two advocates made phone calls and sent emails to their congressional representatives requesting meetings to increase awareness of the challenges facing individuals living with myotonic dystrophy and urge Congress to take action in support of a cure. On Day Three advocates were encouraged to call or email their Representatives and Senators to share their personal stories and urge support for our research funding priorities. 

Day Four was all about reaching out to friends and family members and inviting them to join us in speaking with a louder and unified voice to ensure that we get our fair share of limited federal research funding. We concluded Advocacy Week with a social media campaign we called “Tweet for a Cure.” Advocates from across the country used social media to encourage Congress to support our cause on Twitter, Facebook, and Instagram to continue to build awareness and encourage Congress to support our advocacy priorities. 

“Over the past six years, MDF has succeeded in securing new research DM funding, partnered with the FDA on efforts to accelerate new drug development for DM, and convinced the Social Security Administration to make it easier for families with congenital DM to apply for disability benefits,” said Martha Montag Brown, MDF Vice Chair and NAC member from California. “Our goal is to increase the number of MDF advocates urging Congress to support our priorities every year as part of Advocacy Week.”

Use Your Voice to Change the World for People Living with Myotonic Dystrophy

As Congress advances spending legislation that includes biomedical research funding, we will be closely following these proceedings, and as we identify opportunities for MDF advocates to influence this legislation, we will be urging advocates to follow-up with their US Senators and Representatives. In the fall, Congress is expected to finalize the budget and we will host another MDF advocacy webinar on International Myotonic Dystrophy Awareness Day on September 15th. We hope you can join us for our fall webinar (Register Here!) so we can make our voices heard loud and clear in Washington. Together, we can change the world and improve the lives of everyone living with DM. 

Register Now!

MDF Advocates are the Key to A Cure

Published on Fri, 10/16/2020

According to Merriam-Webster’s dictionary, advocacy is the act or process of supporting a cause or proposal. Since our founding in 2007, Myotonic Dystrophy Foundation advocates have been educating Congress on the need for increased myotonic dystrophy research funding, working with the Social Security Administration to eliminate red tape for individuals with congenital myotonic dystrophy, and leading initiatives with the U.S. Food and Drug Administration to accelerate drug development and better understand the desires of individuals living with myotonic dystrophy.

For over 13 years, MDF advocates from across the country have achieved a remarkable series of victories in our path towards a cure. 

  • Myotonic Dystrophy Research – Since 2007, the National Institutes of Health, the nation’s leading funder of biomedical research, and the Department of Defense Peer Review Medical Research Program (PRMRP), have awarded over $155 million in myotonic dystrophy grants. 
  • Disability Rights – After a year-long advocacy campaign, in 2017, the Social Security Administration added congenital myotonic dystrophy to their Compassionate Allowance Program, which pre-qualifies serious diseases and conditions and provides a faster, streamlined process to apply for and receive disability benefits including health insurance coverage. 
  • Drug Development – MDF hosted the first ever Myotonic Dystrophy Patient-Focused Drug Development meeting in 2016, with FDA senior leadership, to highlight perspectives of patients and caregivers as part of an initiative to drive biopharmaceutical discovery. The Myotonic Dystrophy Voice of the Patient report was producing following this meeting and was delivered to FDA. Since we launched this work, there are now 45 biopharmaceutical companies investigating DM therapies.

These victories have been made possible due to the individual and collective advocacy of our dedicated community. Highlights of these individual efforts include: 

  • In 2014, Kayla Vittek and her mom Lisa Harvey became the first myotonic dystrophy advocates to testify before Congress in support of legislation known as the MD-CARE Act, which has increased federal muscular dystrophy research funding including a dedicated myotonic dystrophy Wellstone Center and related disease prevalence research. 
  • In 2017, Martha Montag Brown, the current MDF Vice Chair, led hundreds of California advocates in a successful effort to have the U.S. Senate add myotonic dystrophy to the PRMRP research program. 
  • In 2019, Tim Haylon, became the first MDF advocate to testify before the House Appropriations Committee. His testimony was instrumental in Congress including provisions in annual spending bills to encourage the National Institutes of Health to make myotonic dystrophy research a higher priority. 

As we look to the future and redouble our efforts to deliver effective myotonic dystrophy treatments and a cure, we need your help. We are inviting every member of the community to become an advocate. Being an effective advocate starts with individuals with myotonic dystrophy, their family, and friends sharing our personal stories. By educating lawmakers and policymakers on the seriousness of the condition, how it impacts daily living, and how individuals with myotonic dystrophy strive to overcome these challenges, we build awareness and relationships in Washington, D.C. Through our advocacy, we urge Congress to embrace our sense of urgency and make critical investments in biomedical research, medical care, health insurance coverage and protections, and therapeutic innovation. 

On the immediate horizon, Congress will complete work on this year’s spending bills following the November election. We continue to closely monitor these efforts and advocate for increased federal funding for myotonic dystrophy research as part of this legislation. As we learn more, we will be inviting MDF advocates to communicate to Congress in support of our research priorities. We will provide updates and sample emails and assist advocates in identifying their Representatives and Senators. We will host quarterly webinars to update the community on our efforts in Washington and provide tools for community members to communicate to our elected representatives.

As we look to next year, in January a new Congress will be sworn in and there will be a president-elect Biden or a second Trump term. These changes will present opportunities to build new relationships in Congress and advance our advocacy objectives. We will be forging ahead with efforts to continue to increase myotonic dystrophy research, pass legislation to strengthen and expand Wellstone Centers, and accelerate efforts to finally bring myotonic dystrophy treatments to market. 

Please consider becoming and advocate and sharing your unique story. It will make a significant difference on our efforts to improve the living of every American living with myotonic dystrophy. 

Click here to watch the Advocacy for DM: Research & Funding session from the MDF 2020 Virtual Conference.

April Advocacy Update

Published on Tue, 05/05/2020

This month’s advocacy update provides a brief report on our ongoing efforts to increase federal funding for myotonic dystrophy research and includes practical information for community members in need of health insurance coverage during the COVID-19 pandemic.

Myotonic Dystrophy Research Funding Update

In late March, Congress passed an unprecedented $2 trillion coronavirus response bill “CARES Act” to provide medical and economic relief during the pandemic. While Congress adjourned for recess following passage of this legislation, the congressional appropriations committees are working behind the scenes to draft budget bills funding the National Institutes of Health and the Department of Defense Peer Reviewed Medical Research Program. We continue our outreach to Congress urging increased federal funding for these vital research efforts with an emphasis on the need for increased support for myotonic dystrophy research. We anticipate Congress will release drafts of these bills in early summer and we will email a legislative action alert in a few weeks so community members can urge Congress to support these requests. Your participation in this campaign is vital so they we can fund important research to accelerate the discover of new treatments and a cure for myotonic dystrophy. Learn more about becomming a myotonic dystrophy advocate!

What to Do If You Don’t Have Health Insurance Coverage?

With record numbers of Americans losing their jobs and often their accompanying health insurance coverage as a result of the pandemic, many individuals and families have questions about health coverage. While the Trump Administration has declined to reopen the open enrollment period under the Affordable Care Act “Obamacare”, there are several options that may help.

  • Under the COBRA law, if you worked for an employer with 20 employees or more, you may be eligible to continue your current coverage. However, this can be an expensive option and you will have to pay the entire monthly premium. This coverage does allow you to see your current doctors and hospitals with the same deductibles and co-pays as you had before. Your health plan must notify you if you are eligible for COBRA and you must decide within 60 days to enroll.
  • If you didn’t have insurance before the pandemic or if you have lost your income because of a job loss, you and your family may qualify for Medicaid coverage. The Medicaid program provides health insurance coverage for low-income Americans. Each state runs its own program, although it’s jointly funded by federal and state governments, so each state program is a little different. You can apply any time and eligibility is based on monthly income, not annual income. Nearly 6 million adults right now qualify for Medicaid but aren't enrolled. Visit your state’s Medicaid program website for more details.
  • Finally, losing a job is a “qualifying event” under the Affordable Care Act so while the enrollment window has closed, you may be able to shop for a new plan through or your state-run exchange. In some states like California, Washington and New York you don't need to have a "qualifying event" like job-based coverage loss or the birth of a child to newly enroll right now. The Kaiser Family Foundation has an easy to use subsidy calculator so you can see what you might pay in premiums for these plans. Your unemployment benefits count as income. According to KFF, more than 4 million Americans who are uninsured would qualify for a subsidized Obamacare health plan that would cost them nothing in monthly insurance premiums.

Coronavirus Aid, Relief, and Economic Security Act

MDF has reviewed the Coronavirus Aid, Relief, and Economic Security (CARES) Act and has identified sections that may be especially relevant to the myotonic dystrophy community. You can read the full summary document here. Below are exerts regarding Cash Payments to Americans:

  • Who qualifies to receive a check and how much will an individual receive? Anyone who filed a tax return this year or last year. Individuals receive $1,200, married couples receive $2,400, and child dependents (under 17) receive $500.
  • What are qualified income levels based off of? There is no qualified income threshold or requirement to receive the rebate. However, the rebate phases out at a 5 percent rate above adjusted gross incomes of $75,000 for single filers, $112,500 for heads of household, and $150,000 for joint filers.
  • Can those collecting Social Security or disability receive a check? Yes, if they filed a tax return this year or last year, or received a form SSA-1099. Otherwise, they need to file a tax return.
  • Will SSA administer the funds to my EBT/Debit card that I receive my SSA benefits through? Our understanding is that IRS is sending out the rebates (via direct deposit or checks)
  • How does an individual claim their check? They do not need to claim their checks (unless they have not either filed a tax return this year or last year) – IRS will send out rebates automatically to their direct deposit or to the address provided on the last tax return submitted.
  • How long will it take for this check to be delivered? Rebates sent via direct deposit will take a few weeks. Rebates sent via checks may take a few months.
  • Will I be taxed on this check? No, rebates are not taxable.
  • Will I be eligible if I haven’t finished filing my 2018 taxes? You need to have filed either a 2018 tax return or a 2019 tax return. If you have not filed either, you will not be eligible. You can file a 2019 tax return now to claim the rebate.
  • Will I be eligible if I have a lien against me, but I am in non-collect status? Yes. Rebates will not be subject to garnishment, except if back child support is owed.

Learn more about COVID-19's impact on the myotonic dystrophy community.

2020 Congressional Leadership Award & Advocacy Update

Published on Fri, 03/20/2020

2020 MDF Congressional Leadership Award – Rep. Adam Schiff

On Thursday, February 27th, in the U.S. Capitol building in Washington, D.C., Martha Brown, the MDF Board Vice Chair, and members of her family, presented the 2020 MDF Congressional Leadership Award to California Congressman Adam Schiff. Congressman Schiff, who serves on the House Armed Services Committee, has been an early supporter of our efforts to add myotonic dystrophy to the Department of Defense Peer Reviewed Medical Research Program (PRMRP) as well as his work securing a visa for Dr. Tahereh Kamali that enabled her begin a three year fellowship at the Stanford University School of Medicine focused on myotonic dystrophy. Dr. Kamali is now hard at work focusing on the central nervous system implications of this disease. 

Congressman Schiff was honored to receive our highest advocacy award and remains committed to our work to improve care and find a cure for myotonic dystrophy. Over the past four years, MDF has grown the number of Senator and Representatives who are committed to our mission. During this period of time we have presented the Congressional Leadership Award to Senators Dianne Feinstein of California and Dick Durbin of Illinois as well as Representative Rosa DeLauro from Connecticut

Updates on the Peer Reviewed Research Program (PRMRP)

Congress launched the Congressionally Directed Medical Research Programs (CDMRP), which includes the PRMRP, in 1992. Myotonic dystrophy research was deemed eligible for federal funding through the program as part of the fiscal year 2018 budget. This success was the result of hundreds of grassroots advocates contacting their Senators and Representatives as well as many in-person meetings in Washington, D.C. Over the past three years, myotonic dystrophy research projects have been awarded over $6 million in new funding. These new projects are advancing our knowledge of the causes of myotonic dystrophy and are advancing the science that will lead to treatments and a cure.

In February, as part of Rare Disease Week, MDF launched a nationwide grassroots advocacy campaign urging Congress to continue to support myotonic dystrophy research as part of the PRMRP. Many individuals, friends and families sent emails and made calls to their Representatives and Senators to increase awareness of myotonic dystrophy and urge their support. This also included a call to action for more support for biomedical research funding supported with federal dollars from the National Institutes of Health.

As community members across the country were sending messages of support to Congress, MDF volunteers and staff were in Washington, D.C. meeting in person with Members of Congress and their staff to deliver the same call to action. We met with congressional staff from both sides of the aisle to press for support. We received numerous commitments of encourage and support from congressional staff including from Senators Feinstein and Durbin.

With the Trump Administration and Congress appropriately focused on efforts to help people through the COVID-19 pandemic, we will continue to closely monitor the congressional budget process and will provide regular updates.  We will also be inviting our advocates to contact their Representatives and Senators in late spring to remind them about our research funding priorities. 

With your continued effort, we can help fund millions of dollars in new myotonic research. Making a difference can be as easy as an email!

UC Irvine Medical School Roadshow

Published on Fri, 03/20/2020

Educating Future Doctors

On March 3, 2020, Loraine Dressler presented to about 40 first and second year medical students at UC Irvine School of Medicine on behalf of the myotonic dystrophy (DM) community. Many of the students come from California but some come from all over the country. They are pursuing medical degrees to become physicians and they all felt very fortunate to have Loraine Dressler and families of the Myotonic Dystrophy support group speak about their experiences. The PALS program at UC Irvine hosted the talk. PALS is a student-initiated public-service program at UC Irvine modeled after the big brother/big sister program. Medical students are paired with children ages 7-17 with a chronic illness/disability in efforts to help the child or adolescent create meaningful relationships and develop coping skills.

Different Appearances Across the Generations

The student group first showed a 10 minute slide presentation that gave a very good overview of DM. Next, Amanda from the student group introduced Loraine and the Orange County Support group. Loraine brought her own family (3 generations) and two other families to represent how different and yet how similar the myotonic dystrophy experience is. Loraine spoke for 20 minutes about her story, including how her brother was diagnosed at 25 years old and died at 47 years old primarily due to esophageal cancer secondary to myotonic dystrophy. She discussed how his physician told him that since his symptoms didn't show until his was an adult, he would probably live a long life with some symptoms, which proved to be very wrong as his symptoms continued to enhance rapidly. He was on a G tube feedings and had numerous aspiration pneumonia challenges and body muscle wasting. Her family was called together when her brother was diagnosed because UCLA wanted to do genetic testing. At that time, there were only muscle biopsies, which could be inconclusive and uncomfortable. Loraine and her sister declined the invitation to do genetic testing as neither of them had symptoms.

A Diagnosis for the Family

Loraine then discussed her more recent history with the disease, starting in 2012, when her younger daughter became pregnant with their first grandchild. She was retaining fluid and the baby wasn't moving. She was sent to a geneticist, who did a family pedigree and ordered an ultrasound. Two hours after the ultrasound and 5 minutes back in the office of the geneticist, three of her family members were diagnosed. The baby was diagnosed with the most severe form of DM called congenital myotonic dystrophy, while her daughter was diagnosed with childhood onset DM, and Loraine was diagnosed with adult onset DM.

Loraine spoke about her muscle tightening experiences throughout her life, including her retirement at age 50 due to not rehabilitating after having bilateral hip replacements. She discussed how challenging it is to care for a child with congenital DM, while having it herself. She discussed all of her grandson's affected bodily systems and spoke about needing a "Team Zen" to work with a person who has a degenerative progressive condition without a cure. Loraine suggested that a social worker who is connected to the community can be the best resource for patients like her family.

Families Share their Stories

A member of the Orange County support group named Emily spoke about her diagnosis, which came after her son was born, when she was showing the neonatologist a family picture. The doctor saw her nephew in the picture and told her he knew that her son was likely born with Congenital DM. Emily had a family gathering and then they discovered that her twin sister, uncle and two nephews had DM. Emily had not been able to conceive and her baby came through IVF. She had not done any testing for DM.

Another group member, Karin, spoke about her struggles with DM including how her diagnosis came after her father-in-law told her that she was dragging her foot and should be seen by a neurologist. Karin has three children, none of whom showed any signs of DM, nor had they been tested. No one in her family had showed obvious signs of DM either.

Put People First

The medical students were encouraged to ask questions and there were thoughtful questions from the audience. The presentation was a great experience overall for everyone involved and validated the experiences of the support group representatives. Loraine’s wish is that the students will remember that every statistic they read represents a person and she hopes that this experience will help inform their schooling and training for years to come.

Learn more about the MDF Medical School Roadshow.

Rare Disease Month: Take Action to Increase DM Research Funding

Published on Fri, 02/07/2020

In 2017, MDF advocates across the country urged their Senators to add myotonic dystrophy to the list of eligible conditions as part of a special Defense Department biomedical research program. The program established in 1999 and called the Peer Reviewed Medical Research Program (PRMRP) has funded millions of dollars in new research in areas including rheumatoid arthritis, spinal muscular atrophy, diabetes and other muscular skeletal disorders. Our 2017 campaign was successful in convincing the United States Senate to add myotonic dystrophy to the PRMRP and this has resulted in scientists being awarded over $6 million for new myotonic dystrophy research projects.

To continue this progress and increase myotonic dystrophy research funding, we need your help!

Please contact your Senators during the month of February which concludes with Rare Disease Day on Friday, February 28th to urge them to include myotonic dystrophy to the PRMRP as part of the Fiscal Year 2021 Defense Appropriations bill. Here’s what you need to do:

  1. To locate your Senators and visit them on the internet to submit your comments, please visit At the top left-hand corner of the website, you will find a dropdown menu of states. From there you can find your Senators websites where you can submit your comments.
  2. Additionally, we have drafted a sample email below that advocates can use to communicate to your Senators. Please take a few minutes to personalize your email with information about how myotonic dystrophy affected you and your family and friends.


Sample Email

February 28, 2020

Dear Senator [Your Senator's Name]:

As we recognize Rare Disease Day, I write to ask for your support for the continued inclusion of myotonic dystrophy on the list of eligible conditions under the Department of Defense Peer Reviewed Medical Research Program as part of the Fiscal Year 2021 Appropriations bill. Myotonic dystrophy is a rare and serious genetic disorder that effects multiple organ systems including the heart, lungs, muscles, brain and gastrointestinal system.

The Senate first added myotonic dystrophy to the PRMRP as part of the Fiscal Year 2018 Defense Appropriations bill and since its inclusion myotonic dystrophy researchers have been awarded over $6 million in new funding. There are no FDA approved treatments for myotonic dystrophy and this research is critically important as part of our efforts to find effective therapies and someday a cure.

As a constituent and volunteer with the Myotonic Dystrophy Foundation, the country’s largest myotonic dystrophy patient advocacy organization, I urge you to ask Senators Richard Shelby and Richard Durbin, the Chair and Ranking Democrat on the Senate Appropriations Subcommittee, to continue to include myotonic dystrophy in the PRMRP.

Thank you for your kind attention to this request and I look forward to your reply.


[Your Name]

Report Out: Community Call January 2020

Published on Fri, 02/07/2020

Written by Northern California Support Group Facilitator, Leslie Krongold, EdD.

To kick off 2020, MDF held a webinar on January 29 for all DM Community & Caregiver Leaders. Over 70 community members registered for the presentation by Leah Hellerstein, Leslie Krongold, and Kevin Brennan. For those unable to attend the webinar, you can download the slides here: Kevin's Advocacy Update and Leslie's DM Family Day at the Park. 

Advocacy Update

The main objective for the webinar was to ignite enthusiasm for two events MDF hopes to capture national participation. The annual Rare Disease Day takes place on February 28th. Kevin Brennan, an advocacy consultant for MDF, presented an overview of Legislative Advocacy 101. Participation from community members for this year’s Rare Disease Day can be achieved at many different access points. For example:

  1. Meeting with your local federal legislative representative;
  2. Participation in a local event with other members of rare disease patient advocacy organizations;
  3. Gathering together as a local community to write letters, make phone calls, and use social media.
  4. Email your senator to keep DM funded in 2020!

In Northern California, MDF is joining with NORD (National Organization for Rare Disorders) and CalRare to hold a public event at the Chabot Space & Science Center in Oakland, California. Last year members from the Northern California MDF Support Group met at Senator Dianne Feinstein’s office in San Francisco to discuss the Orphan Drug Act.

Family Day at Cedar Fair Amusment Parks

The second event MDF is hoping to get nationwide participation with is the DM Family Day at the Park. In April 2019, Leslie Krongold arranged the first DM Family Day at California’s Great America amusement park. This year the event has expanded to include other amusement parks operated by Cedar Fair throughout the United States and Canada.

Currently eleven community members have stepped up to organize a DM Family Day at their local park to be held either this spring or summer:

  1. Buena Park, CA with Loraine Dressler
  2. Northern CA with Leslie Krongold
  3. Shakopee, MN with Liz Naylor
  4. Kansas City, MO with John & Susan Cooley
  5. Charlotte, NC with Mindy Kim
  6. Kings Mills, OH with Suzette Ison & Ted Salwin
  7. Sandusky, OH with Suzanne Perkins & Carolyn Valek
  8. Doswell, VA with Venessa Hudson

If you are interested in participating, please contact Leslie at You can find a Cedar Fair park near you on their official website.


MDF will help all volunteers promote these events to their local DM communities. For more information about holding a Rare Disease Day event in your community, contact MDF at or by phone at 415-800-7777.

Fundraising Campaign and Advocacy Award Created to Celebrate the Life of Kayla Vittek

Published on Thu, 07/18/2019

When Lisa Harvey-Duren, founding executive director of MDF, tragically lost her 13-year-old daughter Kayla Vittek to heart-related complications of the disease in April 2019, she launched a fundraising campaign benefiting Myotonc on Facebook, which to date has raised over $18,000 to help drive Care and a Cure for myotonic dystrophy. A lovely and moving tribute to Kayla’s life can be read here.

To honor and acknowledge Kayla’s numerous appearances to help raise awareness and funds for myotonic dystrophy research and care, and Lisa’s long-time commitment to the foundation and the DM community, MDF will be presenting the Kayla Vittek Memorial Award for Outstanding Community Advocate at the 2019 MDF Annual Conference. Nominations for the award have been submitted by MDF’s advocacy team in Washington, DC to the MDF Board of Directors, which will make the final outstanding advocate selection. We hope you can join us in Philadelphia in September for this award presentation, either in person or via the conference Livestream. Our hearts go out to Lisa and her family for this completely devastating loss, and we are deeply grateful for her ongoing commitment to Care and a Cure.


MDF Advocacy Update - Spring 2019

Published on Wed, 05/08/2019

Over $3 Million in DM Research Funding

MDF has a had a busy few months of activity in Washington, DC – first kicking off the new year by paying a visit to the Congressionally-directed Medical Research Program offices in Fort Detrick, Maryland, in February. MDF CEO Molly White visited with program officers of the Peer-reviewed Medical Research Program (PRMRP), where they discussed the process by which PRMRP grants are awarded. PRMRP leadership also announced during this meeting that myotonic dystrophy research received almost $3.1 million in funding from the Fiscal Year 2018 grant - the first year myotonic dystrophy has been eligible for funding through this program!

MDF also visited with key Congressional offices to thank them for their previous efforts to get access to myotonic dystrophy research funding through the PRMRP and to ask for their continued support to include DM in the program in future fiscal years. MDF Vice-Chair Martha Montag-Brown and Molly presented Congresswoman Rosa DeLauro, the Chairwoman of the Labor, Health and Human Services and Education (LHHS) Appropriations Subcommittee that sets the funding levels for NIH, with the MDF Congressional Leadership Award for her leadership on DM research funding. Chairwoman DeLauro was delighted to receive the award and reaffirmed her commitment to helping the DM community find therapies. Chairwoman DeLauro has been a DM champion for many years and worked to include language in previous appropriations bills that encouraged the NIH to recruit and fund young DM researchers.

MDF Advocate Testifies Before Congress

Working with MDF, Chairwoman DeLauro’s subcommittee also selected MDF patient advocate Timothy Haylon to testify in April at the LHHS Subcommittee Public Witness Hearing. The subcommittee holds this hearing annually, and organizations nationwide related to health, labor, and education may speak before Congress about their priorities in appropriations legislation. Tim was chosen as one of only eight patient organization representatives invited to testify.

After a moving and courageous testimony, which can be viewed here, Chairwoman DeLauro and subcommittee Ranking Member Tom Cole both remarked to Tim that his work as a patient advocate is incredibly meaningful and important, as raising awareness is crucial to helping lawmakers better understand rare diseases and the impact that they have on patients and their families. Tim’s day in Washington also included a visit from his hometown representative, Congressman Jim McGovern, who kindly stopped by the hearing to offer his support. It was an impactful day on Capitol Hill and Tim was an incredible representative of the community.

MDF to Meet with CDC re: Newborn Screening

And finally, in early May, the House LHHS Subcommittee released the Fiscal Year 2020 appropriations bill, which includes language encouraging the Centers for Disease Control and Prevention (CDC) to “review how it can advance education, knowledge, and related outreach activities to foster myotonic screening for newborns.” The bill also requires the CDC to update the committee on this activity in Fiscal Year 2021. MDF is planning to meet with the CDC later this month in its Washington, D.C. office to provide an update on the latest prevalence studies and discuss potential areas of collaboration on newborn screening for myotonic dystrophy, to address the significant diagnostic odyssey that those living with myotonic dystrophy experience.

Get Involved!

MDF will continue to advance newborn screening initiatives, pursue annual PRMRP inclusion, engage lawmakers and make our presence known on Capitol Hill. You can help! Get involved and contact WA, DC MDF representative Kevin Brennan from Bluebird Strategies at

Myotonic Medical School Roadshow

Published on Thu, 04/11/2019

What is the Medical School Roadshow?

MDF designed this volunteer initiative to educate the next generation of medical professionals about myotonic dystrophy in order to improve clinical care and shorten the diagnostic odyssey. MDF is partnering with medical schools to educate students about DM before they graduate and begin clinical work.

MDF Needs You!

We need participation from the people who know DM best: you and those in your family affected by DM. MDF will provide you with training, support in contacting medical schools, and a packet of information and tips. You'll then visit medical schools near your home to speak to second- or third-year students about myotonic dystrophy, including the disease mechanism, symptoms and your personal experience. You can help future doctors learn about DM in the most compelling way possible - by telling your story and providing a real-life picture of the disease in all its variability and whole-body impact.

Get in touch

If you have contacts at medical schools in the US or Canada and are interested in educating future doctors about myotonic dystrophy, we need your help! For more information or to get involved, please contact us at

Get more details on the MDF Medical School Roadshow here.