Thursday, December 1, 2016

Thank you

Thanks to all the generous donors who participated in #GivingTuesday yesterday and chose the Network to receive the funds. We're already busy setting our budget for next year, looking for the best ways to put that money to good use (and to squeeze every penny's worth of value out of it by using volunteer services for most of our activities). 

If you have yet to make an end-of-year gift, we hope you will consider making a donation to the Network so that we can achieve all of our plans and goals in the upcoming years. https://donatenow.networkforgood.org/xlhnetwork?code=donate%20page

Thank you again for your generosity. Stay tuned for some exciting news in 2017 as the work we've done this year, thanks to previous years' donations, comes to fruition with an amazing XLH Day (October 6-7 in North Haven, CT), the launch of the Natural History Study, publication of new educational materials for children, and release of the book of XLH stories (which is still looking for stories; send them to xlhstories@gmail.com). 

We expect 2017 to be something of a breakthrough year, with advances in science, possible FDA approval of pediatric use of KRN23 and the ever-increasingly accepted role of patient groups' involvement with the medical research community. We hope you'll be here, sharing in the good news and supporting our work.

Tuesday, November 29, 2016

It's your turn now

You know how hard we're working to help the XLH community. Now it's your turn to support the Network.

#GivingTuesday is here! Help the XLH Network bring awareness about our disease to medical and patient communities all over the world by giving this holiday season!

Don't forget: in honor of the Network’s twentieth anniversary, donors who give at least $20 will receive an XLH Network sticker!

And don't forget your #unselfie to show your support! Take a picture of yourself holding a sign that mentions the XLH Network, #GivingTuesday, and being #unselfie! Let's take this day to show faces of XLH to people all over the world. And for those of you who prefer not to take #unselfies, we have an "I Pledged" graphic that you can use instead! You'll find it at our official Facebook page: https://www.facebook.com/xlhnetwork.

Now is also a great time to recruit a new member! If you can't give financially, you can give your support. Help one of your friends or family members with XLH become a part of the XLH Network. Ask them to like our Facebook page or join our forum.

Let's make our voices heard this #GivingTuesday! We are our best advocates!

Tuesday, November 22, 2016

Giving thanks

Three things for the XLH (and ADHR, ARHR and TIO) community to be thankful for this year:

1. Hope for the future, with the encouraging data coming out of the KRN23 trials and other research into understanding the metabolic process that leads to phosphate wasting in X-Linked Hypophosphatemia (and the related autosomal conditions, as well as Tumor Induced Osteomalacia.

2. The great attitude of the members of our community. For one example, check out Cheryl's story here: http://ultrarareadvocacy.com/patient-journeys/ And if you want to meet more people who share your experiences (as well as getting the latest information on treatment), mark your calendar now for XLH Day 2017, which will be held in Connecticut on October 6-7.

3. Twenty years of support and advocacy by the XLH Network! And while you're thinking about how much you appreciate the Network, here's a reminder that if you're doing holiday shopping at Amazon in the coming month, you can show your support by enrolling in the Smile program and choosing the Network as your beneficiary. We get a percentage of your purchase, at no additional cost to you! Find out more here: https://smile.amazon.com


Wednesday, November 16, 2016

Giving Tuesday for Twenty Years of Networking

On this, the twentieth anniversary of the XLH Network, we hope you'll remember us on Giving Tuesday, November 29th.

You can donate here: https://donatenow.networkforgood.org/xlhnetwork?code=donate%20page

We'd be particularly grateful for recurring (also known as "sustaining") donations that help to guarantee our long-term viability.

If you can't spare any money, or if you want to do even more than a donation, you can share your appreciation of the Network, encouraging others to consider giving to the Network over on Twitter, using the hashtags #GivingTuesday and #XLHNetwork.

Thank you.

Thursday, November 10, 2016

Ultragenyx filing for regulatory approval of KRN23 in 2017

According to a new press release from Ultragenyx Pharmaceutical, there are plans to file for approval of KRN23 in the US in the second half of 2017. An application is expected to be filed even sooner (around the end of 2016) for Conditional Marketing authorization in Europe.

You can read the entire press release here:
http://ir.ultragenyx.com/releasedetail.cfm?ReleaseID=998128

Also of note in the press release is the following: "Data from the Phase 3 study in adult XLH patients expected in the first half of 2017. The fully-enrolled Phase 3 study will evaluate change in serum phosphorus levels, pain, stiffness, physical function, and safety of monthly KRN23 compared with placebo over 24 weeks in 134 adult XLH patients."

Tuesday, November 8, 2016

Call for UK volunteers

The XLH Network, Inc., is investigating the possibility of establishing country-wide groups affiliated with the Network to address more local issues than a global organization is equipped to handle. 

To that end, board member Oliver Gardiner is looking for volunteers to assist in the creation and operation of an affiliate in the UK (primarily England and Wales). He is looking for individuals (with or without XLH) who could volunteer their skills and expertise to ensure the health and growth of the organisation. You must be over the age of 18. 

To find out more and request an application form, please email: oliver.gardiner at xlhnetwork.org 

Wednesday, November 2, 2016

President's message: Celebrating 20 years of the Network

From the Network's president, Bill Coogan:

As we enter this season of giving and acknowledge twenty years of accomplishments, please help us continue to help you. Donate now:  http://xlhnetwork.org/index.php/donate/


Twenty years ago this summer, Larry Winger created a website to collect information on XLH and then was joined by others to form The XLH Network. The listserv began beta testing in November 1996, and roughly two weeks later, there were subscribers from England, British Columbia, South Africa, and the United States.


A lot has changed since then. We've grown from a community of just a handful of people to a network that reaches thousands of patients and their caregivers. We've gone from not knowing what causes the phosphate wasting to the discovery of the previously unknown hormone, Fibroblast Growth Factor 23 (FGF23). We once viewed the prospect of effective treatment as more science fiction than fact, and now we have a promising new potential treatment on the not-too-distant horizon.


It seems fitting that this anniversary year has been one filled with significant accomplishments. The Network's board has worked hard to maintain all the work of previous years: educating and advocating for the various stakeholders in our community—patients, families and health care providers—by attending medical conferences, sharing what we know about XLH and offering safe places, like our forum and XLH Day, for members to help each other.


Beyond that, though, the anniversary year has also seen some major new initiatives that will come to fruition between now and our twenty-fifth anniversary. The Natural History Study will gather data about the progression of XLH throughout the entirety of patients' lives. For the more personal side of the story, we're collecting stories from our community about living with XLH, for a book entitled Weak Bones, Strong Wills, The Stories of XLH. Several other publications are in the works, including an updated dental brochure and materials to help children of various ages understand their disorder.  


Much of this work is done by volunteers or funded by grants, but there are always uncovered financial costs. The Natural History Study is part of the National Organization for Rare Disorders (NORD) Natural History Program and is paid for in large part by an FDA grant to NORD. Still, the Network will need several thousand dollars a year to insure that the study can continue long enough to provide useful data. Volunteers have donated their time to produce our various educational materials, but we'll still need to pay for their publication. XLH Days are a great experience for those who attend, but again, they can't happen without financial support.