BIRMINGHAM, Alabama – Few people outside his laboratory at the University of Alabama at Birmingham knew or understood what researcher Jerry Thompson did. Over the years, thousands of families across the country relied on Thompson to tell them what was wrong with their children. If […]
Once famously described as “orphan diseases, too small to be noticed, too small to be funded” in the Hollywood drama Lorenzo’s Oil, rare diseases are getting unprecedented attention today among drug manufacturers, who are ramping up research efforts and marketing new medicines that promise fuller lives […]
CAMBRIDGE, Mass., Aug 17, 2011 (BUSINESS WIRE) — Genzyme, a Sanofi company, today announced the first recipients of the Genzyme Patient Advocacy Leadership (PAL) Awards. This global grant program supports non-profit organizations that work on behalf of patients living with lysosomal storage disorders (LSDs), a […]
San Francisco Giants to host Rare Disease Fundraiser for MPS IV – with YouTube sensation Keenan Cahill!
Emil Kakkis loves orphans. The former chief medical officer at BioMarin Pharmaceutical Inc. — the Novato-based poster child for developing treatments for so-called orphan drugs — is lining up cash and personnel for a new venture again aimed at rare diseases. “When you see patients […]