#Amicus Therapeutics announces positive preliminary #results for #Fabry Disease

Amicus, John F. Crowley, Pol Boudes, Fabry Disease, rare diseases, orphan drugs, World Orphan Drug Congress Amicus is currently working on studies for Fabry disease treatment. We just posted about an investigation team led from the University of Massachusetts Amherst that had discovered a genetic interaction that can be a treatment for the rare disease.

Now it is also great news that Amicus, a biopharmaceutical company at the forefront of developing therapies for rare diseases, announced today preliminary results from ongoing phase 2 chaperone-enzyme replacement therapy (ERT) study. Read the complete information here.

John F. Crowley, Chairman and Chief Executive Officer, explained, “These very encouraging data in Fabry patients represent an important first step in validating the potential of pharmacological chaperones to enhance the stability and tissue uptake of enzyme replacement therapy products in Fabry disease."

Amicus' Chief Medical Officer, Pol Boudes will be at the the World Orphan Drug Congress USA 2012 participating in a debate about multiple vs. one global patient registry pros and cons.

He and other experts will discuss about incentives to develop orphan drugs, fast tracking access  and the development of comprehensive patient/disease databases. Don't miss this chance to learn more about strategy, regulation and opportunity for pharma, biotech and investors in rare diseases.

Check the full agenda and register now!