FOSTER CITY, CA--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced positive topline results from its pivotal Phase 3 MARCH study evaluating LIVMARLI® (maralixibat) oral solution in 93 patients with progressive familial...
Emil Kakkis harvesting academic sources for Orphan drug newco Ultragenyx
News | 12. 19. 2011
Ultragenyx Pharmaceutical
Emil Kakkis harvesting academic sources for Orphan drug newco Ultragenyx
By Jennifer Rhodes
Staff Writer
Published on Monday, December 19, 2011
Ultragenyx Pharmaceutical Inc. is taking a traditional approach to Orphan Drugs by in-licensing development-ready substrate and enzyme replacement therapies that have a clear mechanistic rationale and proof of concept in animals for rare and ultra-rare genetic diseases.
Founder, President and CEO Emil Kakkis believes there are a few hundred such diseases with no current treatments.
