Are you bored waiting for the SuperBowl pre-game? Or twiddling your thumbs while waiting for the next commercial to air? If so, please take a few minutes and start to enter your data into the new MLD Patient-Powered Registry. We want to understand MLD better, accurately characterize the MLD patient, family, and caregiver journey, justify […]
Super Bowl Alternative – MLD Registry Read More »
2019 is going to be a very interesting and productive year for MLD therapy development. The trials for both gene therapy and enzyme replacement therapy will be expanded and for the first time are headed towards sites in the United States. A third biotech has publically announced MLD is an active development candidate. We are
5 Biotechs Actively Developing MLD Therapies Read More »
It’s Christmas all over the world … and is usually my tradition, I share some personal thoughts with all of you, my MLD Family. As many of you know, we lost our Darcee to MLD in 1995 and while Lindy is still with us, she is losing the MLD battle bit by bit. Tonight Teryn,
Christmas Message – to the MLD Family Read More »
#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 »
#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 »
We are thankful … for you, your support, and all of the MLD progress this year! Teryn and I are so thankful for the wonderful people we meet as we both live with MLD (Lindy turned 38 this year!) and support families with MLD. We’ve met dozens of new families this year, many in person, as
Always Thankful !!! Read More »
New Clinical Trial for MLD GSK has just launched a Phase 3 clinical trial to study the efficacy of a cryopreserved formulation of their gene therapy, i.e. freezing the modified cells before transfusion. Recruiting of pre-symptomatic late infantile and early juvenile patients will start immediately. This study still requires patients to travel to Milano, Italy
GSK Launches Phase 3 Cryopreserved Gene Therapy Clinical Trial Read More »
#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! #GivingTuesday Read More »
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.
Minnesota at Rare Disease Week on the Hill Read More »
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
A Call for Rare Facts Unity & Consistency Read More »
