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.
Thank you all for your response to my recent request to send letters to the FDA on the Patient-Focused Drug Development initiative last week. Quite a number of you (dozens) responded on behalf of rare disease in general and many on behalf of MLD or lysosomal disease specifically. As I mentioned in my prior post,
PDUFA – 20 Disease Meeting followup … Firestorm – not! Read More »
Welcome to the MLD Foundation’s new blog! We’re excited to share about topics of interest to the MLD, lysosomal disease, leukodystrophy, and frankly, the entire rare disease community. Many of you in the general public and the MLD Family know us from the family support we provide to families affected by MLD … MLD Family
Notifications
