NIH has reissued the CREATE Bio, Blueprint Neurotherapeutics and NeuroNEXT initiatives, along with other initiatives that focus on biomarker development and clinical trial readiness, to support therapy development efforts.
Make a gift to MDF during our Giving Tuesday campaign and double the impact of your donation! Thanks to the generosity of the Haylon and Lord families, MDF will match all gifts made now through December 5th, up to the first $10,000 raised. Give now and significantly increase your support of Care and a Cure for myotonic dystrophy families around the world!
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.
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.
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).