9.30.2013 BIRMINGHAM, Alabama – Few people outside his laboratory at the University of Alabama at Birmingham knew or understood what researcher… Continue Reading
5.17.2013 Once famously described as “orphan diseases, too small to be noticed, too small to be funded” in the Hollywood drama Lorenzo’s Oil,… Continue Reading
1.10.2012 It’s Charity Tuesday and we want to give a shout-out to a new charity called “Courage to Make a Difference” founded by… Continue Reading
9.13.2011 A Rare Disease Patient Perspective I would like to use this blog post to explain why the rare disease community is different, and why it… Continue Reading
8.18.2011 CAMBRIDGE, Mass., Aug 17, 2011 (BUSINESS WIRE) — Genzyme, a Sanofi company, today announced the first recipients of the Genzyme… Continue Reading
San Francisco Giants to host Rare Disease Fundraiser for MPS IV – with YouTube sensation Keenan Cahill!
4.20.2011 No Question, Brian Wilson and Cody Ross are truly dynamite!!! Check out this article and video and pass it on!… Continue Reading
10.27.2010 Emil Kakkis loves orphans. The former chief medical officer at BioMarin Pharmaceutical Inc. — the Novato-based poster child for developing… Continue Reading