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