Advocacy

Update on the MLD NBS RUSP Nomination – and Newborn Screening in General

Leave a Comment / Advocacy, Newborn Screening, Policy, Updates / MLD Foundation / April 23, 2025 / nbs, newborn screening, policy, research, United States

We’re still moving the MLD RUSP nomination forward. We gave/discussed this update at two NBS meetings last week, on an EveryLife Foundation Community Congress ad-hoc call, and in numerous conversations with other patient advocacy groups and bio-pharma companies. We don’t want to leave the MLD community and families out of the update loop, hence this […]

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MLD Gene Therapy Approved in Europe

Advocacy, Awareness, Clinical Care, General, Research, Therapies / MLD Foundation / December 21, 2020 / approval, CHMP, EC, EMA, EU, gene therapy, Libmeldy, MLD

MLD gene therapy approved in the EU

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Your Giving Keeps Us Going – and directly helps families! #GivingTuesday

Leave a Comment / Advocacy, General, Newborn Screening, Research / MLD Foundation / November 27, 2018

#GivingTuesday is Here! Here in the US we celebrated Thanksgiving this past weekend. It’s a time of family, thanks, and giving. Most of us “gave” at the mall on Black Friday or online on Cyber Monday … but now it’s #GivingTuesday, an annual day where the world comes together to give. November is MLD Foundation’s

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Your Giving Keeps Us Going and directly helps families! #GivingTuesday

Leave a Comment / Advocacy, General, Newborn Screening, Research / MLD Foundation / November 27, 2018

Your Giving Keeps Us Going and directly helps families! #GivingTuesday Read More »

Your Giving Keeps Us Going! #GivingTuesday

Leave a Comment / Advocacy, General, Newborn Screening, Research / MLD Foundation / November 27, 2017

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Minnesota at Rare Disease Week on the Hill

2 Comments / Advocacy, Awareness, Education, Policy / MLD Foundation / March 21, 2017 / Alberts, Capitol Hill, Minnesota, policy, Rare Disease Week, RDW, Robertson

Last year when I read Kim Brown’s blog on her experience on the Hill for Rare Disease Week 2016, I told my husband, Trevis, that I wanted to go to Washington DC in 2017 and be an advocate for our precious son, Thomas, who left this earth to become an angel in October of 2015.

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rare disease week Capitol Hill group photo 2017

Our first Rare Disease Week in DC

1 Comment / Advocacy, Awareness, Education, General, Policy / Tony Hodgson / March 21, 2017 / DC, Hodgson, MA, NC, Pierce, Rare Disease Week, RDLA, RDW, Washington DC

At the 2016 MLD Family Conference a conversation with Dean led to a decision to attend the Rare Disease week. It was a first visit for wife Nancy and I to Rare Disease week and a pleasure for us to assist daughter Michelle in attending this year. Last year she had planned to attend but

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A Call for Rare Facts Unity & Consistency

2 Comments / Advocacy, Awareness, Education, General / MLD Foundation / March 4, 2017 / 1 in 10, 7000, advocacy, awareness, facts, statistics

It’s Our Opinion … I presented this poster at the NIH’s Rare Disease Day event earlier this week (2/27/17). In short, to maximize impact and credibility, we are asking all organizations – government, advocacy, industry/pharma, academia, etc. to be consistent with their public sharing of Rare Disease facts. I realize that sometimes we must be very scientific about

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Capitol Hill, BIO, 21st Century Cures, Airbnb, and one more thing … Reflecting on my DC week

Leave a Comment / Advocacy, Awareness, Compassion, General, Policy, Research / MLD Foundation / November 6, 2016 / BIO, Congress, CURES, curesNOW, Eden, FMLD, MLD, policy, RarePolicy

The first rule of blogging is to post good content on a regular basis. The second is to keep your posts short, to the point and to not mix multiple topics into one post. Oh boy, am I in trouble … My 6th trip to DC this year ended reflecting at 35,000′ with a Crater Lake Hazelnut Espresso Vodka courtesy of

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Rare Disease Week in DC … a parent’s perspective

2 Comments / Advocacy, Compassion, Education, General / MLD Foundation / March 14, 2016 / Brown, Capitol Hill, rare disease, RDD, RDW, Washington DC

For those of you who know me, I have been a teaching computer skills to middle school kids for 17 years!. While I love school now as a teacher, I did not enjoy all aspects of school as a student. My favorite classes were Science and Gym! Social Studies, not so much! Shhhhh….. I really

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