The Creating Hope Act was folded into a larger piece of legislation, the FDA Safety and Innovation Act. The bill passed the House last week, and last night was passed by the Senate, and is expected to be signed by President Obama in the next couple of weeks.
How does this affect Armand? I quote a post by NORD (National Organization for Rare Diseases):
Among others, some features of the bill that Saltonstall said were especially important to NORD and the rare disease patient community include:
- enhancing accelerated patient access to new medical treatments
- encouraging the development of Humanitarian Use Devices, or medical devices for small patient populations
- providing for accelerated development of “breakthrough therapies” — or ones that show early promise
- enhanced consultation with rare disease medical experts and
- a rare pediatric disease priority review voucher incentive program
It is this third one that I believe affects Armand the most. There is hope for a possible cure that recently seemed about 10 years away from real human clinical trials. They plan to start working with Tay-Sachs patients much sooner and see how things go from there. As a therapy that “shows promise”, my hope is that we may see human clinical trials much more quickly for GM1 patients.