“There are 7,000 known diseases. We have treatments for only 500 of them. We have work to do.”
That's according to Margaret Anderson, the executive director of FasterCures (a center of the nonprofit Milken Institute),
It's not clear whether XLH falls in the group of 500 with a treatment or the group of 6,500 without a treatment (we do have a treatment, but it's far from a cure), but either way, we all -- policymakers and scientists and patients -- have work to do to find something at least approximating a cure for XLH and other rare diseases, sooner rather than later.
Government isn't the answer to everything, but it can lend a helping hand. The U.S. House of Representatives is working on a program known as the "21st Century Cures Initiative," which you can read more about here. For the next couple weeks, until June 13th, they're soliciting feedback, especially from patients, about "the state of biomedical research and therapeutic innovation for specific diseases and better understand how Congress can help move the ball forward."
Some of the questions being asked by the Initiative are directed at patients, and address issues that XLH patients live with every day. The three that may be of particular interest to XLH patients are:
- What is the state of discovery of cures and treatments for your disease? Are there cures and treatments now or on the horizon?
- How can Congress incentivize, coordinate, and accelerate basic research for diseases we know relatively little about?
- How can we work together to better translate advances in science into safe and effective new therapies for patients?
- What is the financial burden of your disease? How would better treatments and cures help save money for your family and the federal government?
If you're a U.S. citizen and have any thoughts that you'd like to share about encouraging advances in basic science and finding 21st Century Cures, you can email your comments, by June 13th, to firstname.lastname@example.org