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Tag: Emil Kakkis

A total of 13 posts are filed under Emil Kakkis
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
The Food and Drug Administration Safety and Innovation Act (FDASIA) provides major provisions that could help patients with rare diseases.
Historic Legislation Signed by President Obama To Help Patients with Rare Diseases
President Obama signed the Food and Drug Administration Safety and Innovation Act (FDASIA) yesterday.  This new law includes major… Continue Reading
EveryLifeFoundation_new
HR 5651, the Food and Drug Administration Reform Act of 2012
House FDA User Fee Bill Will Unlock Lifesaving Treatments for Rare Diseases Rare Disease Community Applauds House Energy & Commerce… Continue Reading
Sanfilippo_Foundation_ for_Children
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
Rare_Project_Team_Board
‘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
Dr._Stephen_P_Spielberg_FDA
EVERYLIFE Foundation Blog – FDA Commissioner Appoints New Leadership
The Right Person for the Job:  FDA Commissioner Hamburg Appoints New Leadership As we continue to advocate for increased funding and… 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