Building awareness and communities to support and find treatments for Rare Diseases and Genetic Conditions
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 [...]
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 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 [...]
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 [...]
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 [...]
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, [...]
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, [...]
