Advocacy

Myotonic Dystrophy Approved for Important 2019 Defense Funding Program!

Myotonic 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.

Myotonic Secures Compassionate Allowances Designation for CDM

After a year of outreach and education, Myotonic 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

Myotonic 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 Myotonic 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 Myotonic 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.

Myotonic Staff Meets with NIAMS and NINDS

In November, Myotonic 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.

Myotonic 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

Myotonic 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 Myotonic Annual Conference. We designed the meeting to build on the work Myotonic has been conducting with the FDA to define and optimize the regulatory pathway for potential DM therapies. 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. 

Fun Family Tradition Supports DM Community

The Jensen family hosts an incredible crawfish boil each summer to support Care and a Cure. This year’s boil was bigger than ever with San Diego Harley-Davidson as a partner. To date, this event has raised nearly $100,000! Read more here.

Myotonic Making an Impact: Report Back on Myotonic 3.0

Many Myotonic community members are aware of an important, multi-year Myotonic effort -- Myotonic 3.0: Accelerating Drug Development -- because we published an article on the launch of this initiative in early 2015. Read a full report on the first-year results of Myotonic 3.0: Accelerating Drug Development, and the initiatives we are pursuing to promote the development of new DM therapies.  

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