Startup Ultragenyx Snags PiII Program for Growing Rare-Disease Pipeline

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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 […]

A RARE DISEASE PATIENT PERSPECTIVE – Roy Zeighami

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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 deserves a different approach to the approval of treatment. This weekend I started reading Inside the FDA: the politics behind […]

‘R.A.R.E.’ BOARD OF DIRECTORS AND ADVISORS ANNOUNCED

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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 […]

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, […]

A Rare Disease Pioneer Scores $45 Million for New Company – UltraGenyx

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When Emil Kakkis was a researcher at Harbor UCLA developing what would become the first enzyme replacement therapy, he says pharmaceutical companies wouldn’t give him the time of day. The treatment was eventually developed and marketed as Aldurazyme by BioMarin and its partner Genzyme. Kakkis, […]

Ultragenyx – Hereditary Inclusion Body Myopathy (HIBM) New Champion for Hope

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Rocking It For Rare Disease! If anyone can do it, Dr. Emil Kakkis can.  One of the rare disease communities scientific champions (also one of GQ Magazines recognized Rock Stars of Science), Dr. Kakkis comes at his science with a passion and ferver that moves […]

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 treatments for so-called orphan drugs — is lining up cash and personnel for a new venture again aimed at rare diseases. “When you see patients […]