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