Alexion and Cincinnati Children’s Launch Rare Disease Innovation Fund


Alexion Pharmaceuticals, Inc. and Cincinnati Children’s Hospital Medical Center today announced the establishment of a collaboration and fund for the advancement of research in rare disease. Cincinnati Children’s is a recognized center of excellence in pediatric rare diseases. Alexion is a global leader in the […]

Is Roche Going to Do Some Big Rare Disease Drug Deals?

Roche CEO Severin Schwan

So is Roche going to do some rare disease deals or not? Rumors of a possible buyout of Alexion and BioMarin– alternatively dissed and touted– helped make this one of the big questions for deal watchers. Roche CEO Severin Schwan’s answer can now be summarized in one word: […]

MassBio Panel Tackles Why Rare Disease Treatments Cost So Much

David Meeker, right, CEO of Genzyme Corp., speaks at a panel on the pricing of rare disease therapies.

One panel at the MassBio’s annual meeting on Thursday billed under the title, “Rare Disease Therapies: Is The Current Payer Model Sustainable?” probably could have more simply been called, “Why drugs for rare diseases cost so much.” The panel was moderated by Bruce Booth, a partner […]

Faircraft Records/Universal Republic Recording artist Chris Mann and Singer/Songwriter Katrina Parker to Perform at Global Genes | R.A.R.E Project’s Tribute to Champions of Hope™ Benefit

Chris Mann, Nestor Serrano, Jason George and Katrina Parker

FOR IMMEDIATE RELEASE Actors Jason George, Nestor Serrano and Meagan Tandy will Serve as Celebrity Award Presenters; Henri Termeer, Former President and CEO of Genzyme, will Receive Lifetime Achievement Award DANA POINT, CA – August 30, 2012 – Global Genes | R.A.R.E. Project ( today […]

CHI Reports – How Biotech Companies are Shaping the Future in Rare Diseases


Biotech companies are navigating new trends in addressing rare diseases, conditions effecting 200,000 or fewer Americans. Jeremy Springhorn (Alexion Pharmaceuticals), Andrew Curtis (Pfizer), Marc Beer, (Aegerion Pharmaceuticals) and Susan Kahn (National Tay-Sachs & Allied Disease Association), shared their perspectives on the future of new treatments […]

Rare Disease Drugs: A $1 Trillion Market?

Matthew Herper The Medicine Show; FORBES November 2010 Right now drugs for rare disorders are the sweet spot of the biotechnology industry. Treatments for illnesses that affect only hundreds of patients can cost $200,000 or more and have built Genzyme into one of biotech’s biggest […]