research

PDUFA – 20 Disease Meeting followup … Firestorm – not!

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, […]

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Our new blog …

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

Our new blog … Read More »