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.
One family was so inspired by last week’s matching gift that they’ve offered another very generous donation to keep the momentum going! Community members Rick and Paget Haylon will match the next $5,000 donated to MDF through December 10th.
The myotonic dystrophy (DM) community has a strong champion in singer-songwriter Eric Hutchinson. As part of his long-time efforts to support Care and a Cure for myotonic dystrophy, Eric is offering one-of-a-kind fan activities and memorabilia in a new pledge campaign, and a portion of the proceeds will be donated to the Myotonic Dystrophy Foundation (MDF).
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.
Scientists from a consulting firm with considerable experience in evaluation of patient-reported outcome measures evaluated available PROMs for their potential in DM1 clinical trials.