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...
View ArticleChristmas Message – to the MLD Family
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 …...
View Article5 Biotechs Actively Developing MLD Therapies
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...
View ArticleSuper Bowl Alternative – MLD Registry
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...
View ArticleWe have 20/20 Vision for MLD
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...
View ArticleGene Therapy Recommended for Full EU EC Marketing Authorization Review
Gene therapy for early-onset MLD patients passed a huge milestone yesterday when the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion...
View ArticleWe’re thankful – even in 2020!
We heard earlier this week that nearly 4 in 5 people are already looking forward to putting 2020 behind us. The pandemic (family safety, work, school, loss of loved ones, individual and community...
View ArticleGiving Tuesday – It’s Our Day
Did you know that today, Tuesday, December 1st, is Giving Tuesday? After a month of “Black Friday” and yesterday’s “Cyber Monday” frenzy, it’s a day to focus on our loved ones! We aren’t asking for...
View ArticleMLD Gene Therapy Approved in Europe
MLD gene therapy approved in the EU Continue reading → The post MLD Gene Therapy Approved in Europe appeared first on MLD Foundation Blog.
View ArticleMLD Moms are the Best!
We’ve met hundreds of you face-to-face, talked to many of you on the phone for hours, and have met many many more through the Internet. One thing that stands out is the moms. Yes, there are strong...
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