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MDF Advocacy Update - Spring 2019

MDF has had a busy few months of activity in Washington, DC, continuing our efforts to secure research funds, engage lawmakers and make our presence known on Capitol Hill in 2019.

MDF 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. We need you!

Cycling to Drive Awareness and Hope

It was more than five years ago when Jeremy Kleiber went to check on a friend who wasn’t answering his found. He discovered his friend’s body with the man’s three-year-old daughter on his chest, crying for him to wake up.

DM Advocate Glen Wiggans Participates in Federal Grant Review

MDF advocate Glen Wiggans, MD recently participated in the evaluation of research applications submitted to the Peer-reviewed Medical Research Program (PRMRP) sponsored by the Department of Defense. Glen was nominated for participation in the program by MDF. As a consumer reviewer, he was a full voting member of the review panel, along with prominent scientists, to help determine how the $330 million appropriated by Congress for Fiscal Year 2018 will be spent on PRMRP research.

Myotonic Dystrophy Approved for Important 2019 Defense Funding Program!

MDF and our community advocates have again successfully partnered with congressional leaders to include myotonic dystrophy in the Peer-reviewed Medical Research Program (PRMRP) of the U.S. Department of Defense.
 

MDF Secures Compassionate Allowances Designation for CDM

After a year of outreach and education, MDF has successfully convinced the Social Security Administration (SSA) to list congenital myotonic dystrophy (CDM) in its Compassionate Allowances program, significantly easing the application and review process.

DM Voice of the Patient Report Submitted to the FDA

MDF held the first myotonic dystrophy (DM) Patient-Focused Drug Development (PFDD) meeting with key senior leaders from the Food and Drug Administration (FDA) as part of the 2016 MDF Annual Conference in September 2016.

Myotonic Dystrophy Foundation Statement Regarding: American Health Care Act

In March, Republican Members of the U.S. House of Representatives’ Committee on Ways and Means and the U.S. House Committee on Energy and Commerce introduced legislation, entitled the American Health Care Act (AHCA), that would repeal and/or replace major parts of the Affordable Care Act (ACA).

21st Century Cures Act Becomes Law

Thanks to the hard work and commitment of advocates from the MDF community and many others, critical legislation has moved forward that will accelerate the approval of new drugs and medical devices to treat and cure rare diseases like myotonic dystrophy.

MDF Staff Meets with NIAMS and NINDS

In November, Myotonic Dystrophy Foundation (MDF) staff met with the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and the National Institute of Neurological Disorders and Stroke (NINDS) senior leadership and program/policy staff to discuss research opportunities and federal support for myotonic dystrophy (DM). Discussions focused on two areas: the scientific workforce and biomarker and registration endpoint development.

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