This week is the seventh anniversary of the approval of burosumab to treat XLH. That means I’ll have been on it for nine years in July.
It’s been life-changing for me, but I’m not in the mood to celebrate. And all I can think about is that only about five percent of rare diseases have an effective treatment, and now, with the ongoing destruction of medical science in this country, other rare diseases are unlikely to see any progress within my lifetime. And we’re not likely to see the next step for XLH — a true cure instead of a lifelong, expensive treatment — within the lifetime of anyone alive today.
The damage that’s been done to medical research in the U.S. over the course of less than three months is already catastrophic, setting science back an entire generation. And the damage is not affecting just the types of research that might seem frivolous, as it purports to be doing, but is undermining ALL research in this country. Rare-disease research is being particularly hard-hit, since it’s apparently considered wasteful, but also on the chopping block is research into treatment/cures for cancer (both pediatric and adult) and Alzheimer’s, even as we’re on the very edge of breakthroughs for both. Even if the damage is reversed in the next administration, the disruption means we’re unlikely to see significant progress toward treating cancer and Alzheimer’s in our lifetime. And if it’s not reversed quickly, we may not see significant progress in time to treat these conditions in your children’s lifetimes.
Sorry. No uplifting message today. All I can suggest is that if you care about medical research, and you benefited from NIH-supported research (the breakthrough discovery of FGF23 that led to development of burosumab), you should reach out to your federal legislators, and let them know how important rare-disease research is to you. You can find the contact info for your legislators here: https://www.usa.gov/elected-officials
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Please note that the author is a well-read patient, not a doctor, and is not offering medical or legal advice.
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