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Tag: Ultragenyx

A total of 7 posts are filed under Ultragenyx
Ultragenyx
Startup Ultragenyx Snags PiII Program for Growing Rare-Disease Pipeline
Flush from raising more than $100 million, Novato, CA-based startup Ultragenyx Pharmaceutical has stepped up with a new pact to partner on… Continue Reading
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A RARE DISEASE PATIENT PERSPECTIVE – Roy Zeighami
A Rare Disease Patient Perspective I would like to use this blog post to explain why the rare disease community is different, and why it… Continue Reading
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‘R.A.R.E.’ BOARD OF DIRECTORS AND ADVISORS ANNOUNCED
The RARE Project Announces Big Team Additions, Setting the Stage For Continued Success Thirty (30) million people in the US and 350 million… Continue Reading
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:… Continue Reading
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A Rare Disease Pioneer Scores $45 Million for New Company – UltraGenyx
When Emil Kakkis was a researcher at Harbor UCLA developing what would become the first enzyme replacement therapy, he says pharmaceutical… Continue Reading
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Ultragenyx – Hereditary Inclusion Body Myopathy (HIBM) New Champion for Hope
Rocking It For Rare Disease! If anyone can do it, Dr. Emil Kakkis can.  One of the rare disease communities scientific champions (also one… Continue Reading
Former BioMarin exec targets orphan drugs
Emil Kakkis loves orphans. The former chief medical officer at BioMarin Pharmaceutical Inc. — the Novato-based poster child for developing… Continue Reading