February, as the lead-up to Rare Disease Day on February 28th, offers a number of opportunities to take action for your hypophosphatemia (or, more generally, rare disease) community. There are events happening all month, some in-person, some virtual, some local, some worldwide.
I know it can be overwhelming, all the calls for action. But I’m not asking you to do a lot. Just some small indication of support for your favorite rare disease organization. It can, of course, be with money, but I don’t have to tell you that. Instead, I’m here to share a few of the many, many other ways to support them that don’t involve money and only require anywhere from a few seconds of your time to a few hours.
Let’s start with just a few seconds. You know what would REALLY help your favorite rare disease organization, an action that doesn’t involve money and doesn’t even cost much of your time? Following and sharing their social media platforms. For Rare Disease month, pick just one (or you could choose several, of course) organization, and pick one platform that you spend time on anyway, where you’ll support the organization. Then follow that organization on the platform and get in the habit of sharing at least one posting a week for that organization. For just a few seconds more each week, you could also commit to commenting on those posts, even if it’s just saying thank you (although sharing a bit about your relevant experience is even better).
Some possibilities (links are to websites where you can find links to their social media platforms) are:
National Organization for Rare Disorders (NORD): https://rarediseases.org/
European Organization for Rare Disorders: https://www.eurordis.org/
XLH Network: https://xlhnetwork.org/
International XLH Alliance (you can also find links to their member organizations here if you prefer to follow them individually instead of as a group): https://xlhalliance.org/
Canadian XLH Network: https://canadianxlhnetwork.org/
If you have a couple hours on just one day, consider attending a webinar for rare disease day or rare disease advocacy:
There’s Quinnipiac University’s Rare Disease summit on February 25th. https://quinnipiac.digication.com/rare-disease-day-symposium-2022/home Or find another event, possibly including one near where you live, at NORD’s website (scroll down to the event finder section): https://www.rarediseaseday.org/
If you have a whole day available (either on February 28 or later), register for the National Institutes of Health Rare Disease Day virtual event: https://ncats.nih.gov/news/events/rdd Once you’re registered, you should be able to stream the recording later if you can’t watch it live.
If you have an ongoing hour or two per month (or week!) to commit to patient advocacy, consider doing one of the items above, and keep an eye out for opportunities discussed in those social media postings or the events you attend, and sign up for those volunteer opportunities. I also strongly encourage you to subscribe to the newsletter for the EveryLife Foundation (founded by the founder of Ultragenyx): https://everylifefoundation.org/newsletter-signup/ They offer good training for patient advocates, and are a good resource for learning about advocacy opportunities.
If you’re ready to commit even more time to patient advocacy, I’d recommend attending either NORD’s rare disease summit or the Global Genes Patient Advocacy Summit. I didn’t even know what was possible with advocacy until I attended the NORD summit a few years ago and watched some of the Global Genes events online. Patient groups are doing amazing work these days, from the support you’re familiar with to sponsoring scientific research and even clinical trials!
I know we’re all busy and are facing demands from a myriad of directions, so I understand there may be tight limits to the amount of advocacy you can do. Still, if you’ve ever felt grateful to a patient group or advocate, please consider honoring their work by taking the plunge (or diving deeper) into patient advocacy this month or this year. We’re at a turning point in so many battles for better treatments and access right now, so we need the community’s support more than ever. It really doesn’t have to be a major time commitment. We say that “together we’re stronger,” and that applies to advocacy too; the more voices who join in, the better chance we’ll be heard.
Advocacy isn’t even a purely philanthropic act. In my experience, advocating on behalf of others just makes me feel good, especially on days when my XLH (or some other aspect of life) is really getting me down. Advocacy gives me back a tiny bit of the sense of being in control of my life. I may not be able to run a marathon these days (okay, I never could do that), but I can do something small like contact my legislator to ask them to support the Ensuring Lasting Smiles Act, or to establish a Rare Disease Council in my state, and groups like NORD, Global Genes, and the EveryLife Foundation can guide you on how to do those things.
And for anyone who has a child or grandchild (and the prospect of additional generations) with XLH, do it for them. I believe that part of the reason why the clinical trials for burosumab went so smoothly (beyond the excellent work by the principal investigators and their hard-working staffs), is that patients all over the world were already connected and fairly easily recruited. That wouldn’t have been the case if not for a few patient advocates deciding to start a listserv some twenty-six years ago. We’ll need that sort of preparation for the next big leap in treatment (a cure!) and access to state of the art treatment, and you never know what seemingly little project today will lead to a major advance that will improve the lives of your children/grandchildren/descendants.
Regardless of why or how much you do, please do something this month to advocate for rare diseases or to learn how to be an advocate. If you’re wondering if I’m backing up my words with my own action, my goal for this month is to learn more about gene therapy, so I can share with you what I’ve learned about it and how it applies to us.
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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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