BioMarin Gets U.S. Panel’s Backing on Rare Disease Drug

Biomarin

BioMarin Pharmaceutical Inc. received the backing of U.S. advisers for a drug to treat a rare metabolic disease that can affect growth. The drug, Vimizim, should be approved for Moriquio A syndrome, which can cause joint deformities and contractures, a panel of advisers to the Food and Drug […]

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: […]

Startup Ultragenyx Snags PiII Program for Growing Rare-Disease Pipeline

Ultragenyx

Flush from raising more than $100 million, Novato, CA-based startup Ultragenyx Pharmaceutical has stepped up with a new pact to partner on a rare bone-disease drug from Japan’s Kyowa Hakko Kirin. Under the guiding hand of BioMarin veteran Emil Kakkis, Ultragenyx will step in to co-develop KRN23, an antibody designed […]

Nimbus Discovery, Shire to Co-Develop Oral Rare Disease Drugs

nimbus_Discovery

Nimbus Discovery splashed onto the scene in 2011 with a big-name investor group that included Bill Gates and a plan to discover drugs with the help of computer software. On Tuesday, that idea turned into a potentially big partnership with Shire  to help conquer currently untouchable rare […]

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 (www.globalgenes.org) today […]

Global Genes and R.A.R.E. Project Announce Tribute to Champions of Hope™ Gala to Honor Rare and Genetic Disease Pioneers, Innovators and Advocates

Henri_Termeer_Rare_Disease_Drugs_Genzyme

Henri Termeer, Biotech Legend and former President and CEO of Genzyme, To Receive Lifetime Achievement Award; Nominations Now Being Accepted For Medical Care and Research & Science Award Categories DANA POINT, CA – June 27, 2012 – Global Genes (www.globalgenes.org) and the R.A.R.E. Project are […]

Wall Street Journal 'Health Blog' Reports – GETTING MORE DRUGS APPROVED FOR RARE DISEASES

By Amy Dockser Marcus A new paper offers a possible road map for getting more drugs approved to treat rare diseases. One key recommendation: improving access to the FDA’s accelerated approval pathway, originally developed to quickly get treatments to patients with life-threatening diseases. The paper, […]