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As our legislators headed home for their August break, Rare Disease Legislative Advocates got busy. They contacted everyone on their contact list and asked them to participate in In-District Lobby Days. I simply registered on their website and notified them of my availability so that they could schedule appointments for me with my Senator and Congressman.

I visited Senator Kirsten Gillibrand’s office as a representative of our group, and was joined by others lobbying for their respective rare diseases. We met with Senator Gillibrand’s senior advisor, who spent over half an hour with us listening to our stories and our “asks.” I highlighted the need for sponsorship for the Medical Equity Act, which would mandate coverage of metabolic formulas and low protein foods.

When I met with Congressman Engel, it was me alone with him and his Chief of Staff! We shared family stories, talked a bit about politics in general, and then got down to my specific concerns. Congressman Engel is a member of the Rare Disease Congressional Caucus, so I knew I had a supporter sitting in front of me. He asked me to tell him about MSUD, which I did in great detail. I then told him about this specific bill and asked him to consider co-sponsoring the legislation. Of course he said he needs to read the bill first, but did assure me that this was a piece of legislation he was likely to support. I also emphasized the need to fully fund the FDA and NIH, which he agreed was vital to the health of our nation. Unfortunately I forgot to ask for a picture!

Advocating with my elected officials was easy to do and, truthfully, fun! When people hear our stories, they want to help. I urge every one of you to try it!

Fundraising

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Updates To Nutrition Management Guidelines

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NBS-MSUD Connect: Advancing MSUD Research And More

NBSCONNECT.ORG

Rare disease registries have received attention in recent years because of the many ways in which they can benefit the rare disease community.

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From The Chairman’s Desk

On July the 21st the MSUD Board of Directors traveled to Bethesda, Maryland for our annual Board meeting. We stayed at the Hyatt Regency in Bethesda.

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MSUD Advocacy Update

Healthcare and issues facing the rare disease community have been at the forefront of national conversations over the past 6 months.

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Metformin As A Possible Therapeutic Agent In The Treatment Of MSUD

The MSUD Family Support Group has provided funds to Buck Institute for its preliminary studies investigating metformin

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A Child's Life

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