Policy

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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We have 20/20 Vision for MLD

Leave a Comment / Awareness, Clinical Care, Compassion, Education, General, Newborn Screening, Policy / MLD Foundation / December 31, 2019

2020 will bring new clinical and therapeutic care to the MLD community. When we started serving MLD Families in 1999 – two decades ago – we had no idea how challenging and time-consuming it would be to improve clinical care and disease altering therapies. Along with your support, we’ve done a lot of heavy and

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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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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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Mld Family Conference™ – Register Now!

Leave a Comment / Clinical Care, Education, General, Newborn Screening, Policy / MLD Foundation / May 19, 2015 / Biffi, CBD oil, conference, family conference, marijuana, medical marijuana, newborn screening, RUSP, shire

Registration for our 2105 MLD Family Conference in Newark Delaware is underway and closes in just a couple of weeks. If you are a MLD Family we’re anxious to have you register. Many of your MLD Family will be there to meet, share, and socialize with. Please bring your MLD loved one with you – we not only want

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Join our call for OPEN Natural History Studies

Leave a Comment / Advocacy, General, Policy, Research / MLD Foundation / March 26, 2014 / collaboration, manifesto, natural history, openNHS, patient driven

A new Natural History Study (NHS) for MLD was launched a few weeks ago. We have taken the bold step of recommending that families NOT participate in this study … for now: It is our belief that all natural history studies be OPEN access, meaning the study data be maximized by being made available to other researchers from other academic

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Say it’s not true … New Zealand proposes NO access to an “effective therapy” for a rare disease

Leave a Comment / Action Alert, Advocacy, Policy / MLD Foundation / July 21, 2013 / Alexion, eculizumab, LDNZ, New Zealand, Pharmac, PHN, rare disease, Soliris

Has my Sunday morning coffee not kicked in? Am I reading this wrong? Tell me it’s not true … Alexion has an “effective therapy” for paroxysmal nocturnal haemoglobinuria New Zealand is proposing to decline access to therapy for its citizens … not because it does not work – they acknowledge it is an “effective therapy” – rather, they

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Newborn Screening – Should a Viable Therapy be a Requirement for a NBS?

Leave a Comment / Advocacy, Newborn Screening, Policy, Research / MLD Foundation / May 16, 2013 / nbs, newborn screening, panel, Patterson, RUSP, SACHDNC, summit, viable therapy

The requirements for adding a Newborn Screen (NBS) to the RUSP (Recommended Uniform Screening Panel by the SACHDNC/DACHDNC (Secretary’s Discretionary Advisory Committee on Heritable Disorders in Newborns and Children) consists of four primary criteria: An acceptable treatment protocol in place that changes the outcome for patients diagnosed early with the disease An understanding of the condition’s

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Rare Disease Advocacy – Behind the Scenes

Leave a Comment / Advocacy, Awareness, Policy, Research / MLD Foundation / May 13, 2013 / FDA, Jonah's Just Begun, policy, research, shire, Wood

I ran across this article a few minutes ago. It’s a great insight into the challenges those of us working in rare disease advocacy working encounter. Every person and agency mentioned in this article is someone we at the MLD Foundation regularly come into contact with as we work on behalf of those with MLD.

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