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 […]

Unlocking Lifesaving Treatments Act – ULTRA Act of 2012

Emil_Kakkis_Rare_Disease_Ultragenyx

In December 2011, U.S. Representatives Cliff Stearns (R-FL) and Ed Towns (D-NY), two senior members of the Energy and Commerce Committee, introduced bipartisan legislation to help spur the development of treatments for very rare diseases. The Unlocking Lifesaving Treatments for Rare Diseases Act of 2012, […]