What we really like about this bill is it's something that can happen sooner rather than later, and it can be absorbed and included into whatever The FDA is doing to improve, as well, looking ahead to the next near, or next five years, just really continuing the conversation on rare disease.

Melissa Goetz

It began with our frustrations of FCS, but as we were talking with other rare disease groups and sharing our frustrations and concerns with them, we realized that this was not only an FCS-specific issue, a lot of other rare disease groups were struggling to get treatments for The FCS community as well.

She was vomiting, they drew her blood, and it was that strawberry milkshake-colored pink, it was a very difficult time emotionally for me being postpartum and having her diagnosed with a rare disease.

Some of these drugs are going to require high level monitoring physicians, and I think there can be an idea that that level of monitoring can be burdensome for the parents, but this is something we've been talking about with The FDA -- most patients welcome that level of monitoring because it means they're going to potentially be healthier and have better care.