Amicus Announces Positive Data in First Fabry Phase 3 Study

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Amicus Therapeutics, a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced positive 12- and 24-month data from its first Phase 3 study (Study 011) of the oral small molecule chaperone migalastat HCl (“migalastat”) monotherapy in Fabry patients with amenable […]

JPMorgan Dad Turns Biotech CEO in Quest to Save his Son


Ilan Ganot, a hedge fund banker at JPMorgan Chase & Co. (JPM), left his life in finance to create a new business model aimed at developing drugs more quickly. His son’s life may depend on his success. Since his 2-year-old was diagnosed a year ago with […]

Lance Legislation Designed to Help Patients with Chronic Diseases and Disabilities


Lawmaker joins noted rare disease advocates in effort WASHINGTON, D.C.– Congressman Leonard Lance (NJ 07) today announced he has reintroduced legislation that seeks to promote the development of meaningful treatments for patients with chronic and rare diseases. The bipartisan “Modernizing Our Drug and Diagnostics Evaluation and Regulatory Network […]

John Crowley Talks About the Fifth Prescription Drug User Fee Act


On the last day of the World Orphan Drug Congress, John Crowley, Chairman and CEO, Amicus Therapeutics provided an inspiring overview of how the Fifth Prescription Drug User Fee Act (PDUFA V) and new government policies may affect the orphan drug industry. From a historical […]

The Importance of Patient Initiated Research and Virtual BioTechs

Eileen O'Brien, Siren Interactive's Director of Search & Innovation.  Eileen has more than 16 years of digital healthcare marketing experience and is considered an opinion leader on social media

Sharonne N. Hayes, MD, from the Mayo Clinic, recently authored a blog, “The potential of patient initiated research in studying rare diseases,” where she described her experience with the rare disease SCAD–spontaneous coronary artery dissection. Katherine Leon had connected with other SCAD patients via the WomenHeart Inspire […]



The RARE Project Announces Big Team Additions, Setting the Stage For Continued Success Thirty (30) million people in the US and 350 million people worldwide are affected by rare disease.  Rare disease affects more children and their families then AIDS and CANCER combined worldwide.  The […]

Rare Disease Day 2011: Research, Action and Impact

Updates from MarbleRoad for Rare Disease Day 2011, including events, publications, and a quick shout-out for those fighting rare kidney disease.