Patient advocates have amazing power. The 1983 Orphan Drug Act enabled an entire industry treating rare diseases. However, the fact is it wasn’t enough. A huge portion of the 7000+ rare diseases are still without treatment.
This upcoming year, a number of patient groups have pulled together to endorse ULTRA, the Unlocking Lifesaving Treatment Act. It will remove key barriers to enabling accelerated approval for rare genetic diseases. We believe it will unleash a huge amount of biotech investment and result in many new treatments for those suffering from the rarest of diseases.