Wednesday, February 21, 2018

Questions about burosumab?

You know that the new treatment for XLH, the antibody to FGF23, and known as burosumab, is under consideration by the U.S. Food & Drug Administration and by the European Medicines Agency with (we hope) approval due sometime in the next few months.

You must have questions about what happens after the approval. We're working on some resources that will answer as many questions as possible about burosumab and how to discuss it with your doctor, your insurer and your pharmacist.

We've been wracking our brains to anticipate all your questions, starting with the belief that they're the same questions we have ourselves. But everyone's experience with XLH is different and everyone has different concerns, so we need your help to make sure we haven't missed any important topics.

So here's your chance to tell us what you've been wondering about for the future of XLH treatment. We won't be answering your questions right now (and we can't advise you on whether treatment is right for you or your family specifically), but we'll try our best to make sure that the materials we'll be publishing this spring will contain enough information to answer all your non-patient-specific questions.

You can send your questions privately to info@xlhnetwork.org, or post them in a comment at our official facebook page, https://www.facebook.com/xlhnetwork/

Wednesday, February 14, 2018

Valentine's Day hearts

How's your heart?

While some early research (of limited value due to the tiny number of patients studied) suggested a possible correlation between XLH and heart disease, some very recent research suggests that FGF23, the hormone that XLHers have in excess, may actually prevent/slow heart disease! https://phys.org/news/2018-01-anti-aging-hormone.html

The research is far too preliminary to be able to draw any conclusions about the XLH population's heart health. We hope our natural history study, launching later this year, will help us understand more about the incidence of heart disease in the XLH community.

In the meantime, take good care of your heart today and every day of the year!

Monday, February 12, 2018

Rare Disease Day at Quinnipiac University

Have you signed up to be a Rare Disease Day champion? There are lots of ways you can do that! Get all the details you need here! 
https://xlhnetwork.networkforgood.com/projects/45764-rare-disease-day-champions

And while you're making plans, consider attending Rare Disease Day (a day early on February 27) at Quinnipiac University. Our president, Bill Coogan, will be in the audience and the public is welcome.

Wednesday, February 7, 2018

Save the date: XLH Day 2018!

This year's XLH Day on Columbus Day weekend (October 5-7) in the Baltimore/Washington, DC area is going to be bigger and better than ever!

We're adding a one-day pre-event called "Symposium on Hypophosphatemia: past, present and future," which will focus on the experiences and challenges for adults living with XLH and related hypophosphatemias, including the autosomal versions and Tumor Induced Osteomalacia. It's an opportunity to discuss these issues in a structured environment, with representatives of the medical community and the U.S. Food & Drug Administration in the audience. The event will be videotaped and transcribed for sharing with the FDA and the medical community (including your clinicians!).

To read more about events like this (which in government-speak are called externally-led Patient Focused Drug Development meetings, or PFDDs), check out this site (and don't be intimidated by all the jargon): https://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm453856.htm

We'll have lots more information for you over the coming months, but for now, we're hoping you'll put the dates on your calendar and start making plans to attend. It's going to be fabulous, and we want you to be part of it.

Thursday, February 1, 2018

Countdown to Rare Disease Day

We are officially kicking off our Rare Disease Day Campaign today!

The theme of this year’s Rare Disease Day is "Research." We all know how important research is for the XLH community, and as we gear up for a potentially life-changing 2018, we hope you will become a Rare Disease Day champion.

You can download our toolkit for ways to participate. Have your city or town declare February 28th as Rare Disease Day! Tell your story! Host a meetup!

Or you could participate in #HandprintsAcrossAmerica with the information here: http://rarediseaseday.us/get-inv…/handprints-across-america/

You can also help us raise funds on Rare Disease Day by creating a personal campaign and letting everyone know why the Network is important to you.