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. …
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