A very interesting question came up today on the MLD Family Discussion List™ (a private list we run for MLD primary caregivers). I thought it might inspire some thinking so I am sharing my answer here as well: I have a question and I don’t know if anyone has an answer but I am wondering if anyone […]
Are normal enzyme levels enough to stabilize MLD? 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 »
We are pleased to share that a Phase I/II Intracerebral Gene Therapy clinical trial for MLD is now recruiting late infantile MLD patients. Dr. Patrick Aubourg and Dr. Caroline Sevin are the co-Principal Investigators. We have posted complete details of the trial, including inclusion criteria, here. This trial is based on many years of work in
Intracerebral Gene Therapy Phase I/II Clinical Trial for MLD Read More »
I was pleased to be the organizer and host for the RARE Project | Global Genes RARE Patient Advocacy Summit on September 29th, 2012. The day-long event with 140 in attendance and over 120 viewing via a live webcast. Videos of the event are available below for viewing.
RARE Patient Advocacy Summit – 2012 Read More »
This list was prepared using data supplied by the NIH’s Office of Rare Disease Research in late 2011. Please note that your medical providers and insurance payors probably have their own lists of diseases they think are rare – this list is for quick reference only. Quick Jump: A B C D E F G
List of Rare Diseases Read More »
