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 […]
Mld Family Conference™ – Register Now! Read More »
A meeting of researchers, clinicians, industry and academia was convened by the MLD Foundation on June 24th in Washington, DC to discuss the openNHS Manifesto we wrote about in this blog post. Since a NHS is not a therapy, NHS participants have historically be giving time, energy, and effort, not to mention exposing their MLD loved ones to occasional invasive
openNHS Manifesto – Meeting Report Read More »
The Enzyme Replacement Therapy HGT-1110 Phase I/II clinical trial has been fully recruited. Patients are participating from three continents; Europe, South America, and Australia. The primary purpose of a Phase I/II clinical trial is to study safety and to establish a target dosage for further study. The trial lasts 40-weeks so the last patient should complete their
ERT Phase I/II Clinical Trial Recruiting Completed Read More »
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.
Rare Disease Advocacy – Behind the Scenes Read More »
