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Myotonic Medical School Roadshow

Myotonic 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!

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 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, 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.

MDF Honors Community Leadership Award Winners

Congratulations to our Community Leadership Award winners: Loraine Dressler, Carolyn Valek and Eric & Taylor Jensen! Click here to learn more about the amazing work they do for Care and a Cure.

DM PFDD Meeting - Bringing Your Voice to Therapy Development

MDF held the first myotonic dystrophy Patient-Focused Drug Development meeting with key senior leadership from the Food and Drug Administration (FDA) as part of the 2016 MDF Annual Conference. The meeting, the first Externally-Led PFDD meeting and the largest PFDD meeting held to date with over 200 attendees in the room and online, is helping change the way the FDA understands DM, the DM community and the approach to regulatory review for DM therapies. 

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