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 […]

Historic Legislation Signed by President Obama To Help Patients with Rare Diseases

The Food and Drug Administration Safety and Innovation Act (FDASIA) provides major provisions that could help patients with rare diseases.

President Obama signed the Food and Drug Administration Safety and Innovation Act (FDASIA) yesterday.  This new law includes major provisions of importance to the rare disease patient community and creates several changes to FDA policy including a provision to empower the FDA to accelerate approval […]

HR 5651, the Food and Drug Administration Reform Act of 2012

EveryLifeFoundation_new

House FDA User Fee Bill Will Unlock Lifesaving Treatments for Rare Diseases Rare Disease Community Applauds House Energy & Commerce Committee for User Fee Reauthorization Legislation that Spurs the Development of Treatments Thursday, May 10, 2012, WASHINGTON, DC—– Today the House Energy & Commerce Committee […]

A RARE DISEASE PATIENT PERSPECTIVE – Roy Zeighami

Sanfilippo_Foundation_ for_Children

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

Rare_Project_Team_Board

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 […]

EVERYLIFE Foundation Blog – FDA Commissioner Appoints New Leadership

Dr._Stephen_P_Spielberg_FDA

The Right Person for the Job:  FDA Commissioner Hamburg Appoints New Leadership As we continue to advocate for increased funding and improved specialized review divisions at the FDA, we are encouraged by the recent positive action coming from the Commissioner’s Office.  Commissioner Hamburg has reorganized […]

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, […]