MLD Gene Therapy Approved in Europe
MLD gene therapy approved in the EU
Advocacy
Your Giving Keeps Us Going – and directly helps families! #GivingTuesday
#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
Your Giving Keeps Us Going – and directly helps families! #GivingTuesday Read More »
Your Giving Keeps Us Going and directly helps families! #GivingTuesday
#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
Your Giving Keeps Us Going and directly helps families! #GivingTuesday Read More »
Your Giving Keeps Us Going! #GivingTuesday
#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
Minnesota at Rare Disease Week on the Hill
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.
Our first Rare Disease Week in 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
A Call for Rare Facts Unity & Consistency
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
Capitol Hill, BIO, 21st Century Cures, Airbnb, and one more thing … Reflecting on my DC week
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
Rare Disease Week in DC … a parent’s perspective
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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MLD Newborn Screening Pilot Study – Announcement
Today is Rare Disease Day. It’s an appropriate day to announce we have come one step closer to a MLD Newborn Screen! The pilot study to test the newly developed MLD Newborn assay/technique on current newborn babies born in Washington state will be starting within the next three months once testing lab equipment is in place.
MLD Newborn Screening Pilot Study – Announcement Read More »