There are a variety of ongoing issues that face the MSUD and greater rare disease community. While we push forward to improve the lives of those with these diseases, here is a quick update on the some recent advocacy issues that are facing the rare disease community:

  • The Senate Authorizes Medical Foods Coverage for the Military in National Defense Authorization Act. This bill allows for medical foods to be covered under the TRICARE health care program for Department of Defense and Military families. The bill will need to be harmonized with the House version passed a month earlier.
  • The Senate delayed their vote on the Senate Innovations for Healthier Americans Initiative, also known as the Senate Cures Legislation. Senate Cures is the companion legislation to the 21st Century Cures Act, which passed the House last year. The legislation includes billions of dollars to help the rare disease community, including new funding for critical research at the National Institutes of Health (NIH) and to accelerate drug approval at the FDA, and several other provisions. Rare Disease advocacy groups will continue to push for this legislation to pass once the Senate is back in session in September.
  • The State of Illinois is waiting for a bill to be signed by the governor that would establish a Rare Disease Commission that would give patients a voice in the state government. The bill would also provide educational resources for elected leaders on critical issues related to access, coverage, and the diseases themselves.
  • A Rally for Medical Research Day will be held on September 21, 2016 on Capitol Hill. Over 300 organizations will participate to urge members of Congress to make funding for the National Institutes of Health (NIH) a national priority and raise awareness about the importance of continued investment in medical research. To sign up for the “Rally” reception or the “Rally Hill Day”, visit
  • There are several patient groups in Oregon seeking to reduce the out-of-pocket drug costs for patients. The Oregon legislature recently created a Prescription Drug Cost Working Group who met for the first time this past June. The National Organization for Rare Disease (NORD) is supporting policy recommendations, which encourage the State Working Group to address cost barriers and other issues that impede patient access to medicines. If advocates are interested in learning more about the Working Group and these issues, contact Tim Boyd at This email address is being protected from spambots. You need JavaScript enabled to view it.

Larger issues like the Medical Foods Equity Act and better timeliness with Newborn Screening are works in progress. If you are interested in advocating for MSUD in your state or federal governments, please contact me: This email address is being protected from spambots. You need JavaScript enabled to view it..


In the 2016 survey of the membership of the MSUD Family Support Group, research for improved treatments and potential cure was rated “most important” by 90% of the responding members.

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A Child's Life

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