Tuesday, November 25, 2014

Giving Tuesday

This year has been a busy one, with lots of firsts, like our recent Midwest XLH Day. Now it's time for another first: our first year participating in #GivingTuesday.

If you've never heard of the event, here's how the official website describes it: "We have a day for giving thanks. We have two for getting deals. Now, we have #GivingTuesday, a global day dedicated to giving back. On Tuesday, December 2, 2014, charities, families, businesses, community centers, and students around the world will come together for one common purpose: to celebrate generosity and to give."

If you appreciate the work the Network has been doing, here are a few ways you can give back on #GivingTuesday.

1. Give money. We're working toward a more stable future for the Network, and that takes financial resources. While we love large donations, they're not the only ones that make a difference. When a large percentage of an organization's members donate, even if the individual amounts are small, that evidence of our members' support can improve our chances of obtaining grant funding. No matter how much or how little you can afford, every dollar makes a real difference. Here's the information on how to make a donation: http://xlhnetwork.org/index.php/donate/

We understand that not everyone is in a position to make even a small financial contribution, but there are other ways to give back.

2. Give knowledge. You know about the Network and our mission, so tell someone else about us. Maybe there's someone in your extended family who has XLH, but hasn't joined the Network yet, and you could encourage them to join. Maybe your doctor or dentist treats other XLH patients, but doesn't know about the Network or all the resources we offer. In that case, you could print out some of our brochures (medical here:  http://xlhnetwork.org/files/7813/9680/2734/XLH_brochure.pdf, and dental here http://xlhnetwork.org/index.php/what-is-xlh/dental-flyers/) and ask the medical provider to share the information with other patients.

3. Give hope. If you've benefitted from membership in the XLH Network, and are excited about the recent advances in research, tell someone. #GivingTuesday is a perfect opportunity to announce your support of the XLH Network. If you're not already connected with our social media platforms, you can like our official facebook page (facebook.com/xlhnetwork) or follow us on Twitter (@XLH_Network). Then you can share our posts/Tweets, and add your comments to let your followers know what the Network means to you. If you're not sure what to say, keep it simple: "I support @XLH_Network on #GivingTuesday and throughout the year."

We are thankful for the support of all our members in whatever form it takes. We hope you'll join us in raising awareness about the XLH Network as we embark on our first #GivingTuesday campaign. You can make a difference: Give money. Give knowledge. Give hope.

Wednesday, November 19, 2014

Helping the researchers

We're coming up on the traditional time to think about what we're grateful for.
We'd like to suggest that you think about some people you probably haven't met: the researchers working on a cure for XLH. They need our help.

Until recently, research was done without much consideration of the patients' day-to-day needs. While a great deal of good work was done, it's not the only way to proceed and may well not be the most efficient way to proceed. Finally, patients are being given the opportunity to contribute to the research in a variety of ways.

At the moment, we in the XLH community have three opportunities to participate in such patient-centered research.

The newest one is the Rudy study, now open for registration to all adults and children with XLH who live in the UK. The aim of the study is to improve understanding of all aspects of rare bone diseases with the aim of developing new tests and treatments to improve patients’ lives. Rudy is led by a research team at the University of Oxford which is funded by the NIHR Rare diseases of Bone, Joint and blood and the Oxford NIHR Musculoskeletal Biomedical Research Unit, University of Oxford. If you are interested in finding out more including how to register please check the library on their website www.rudystudy.org.

The second opportunity i s coming to a close just as the Rudy study begins. It's the Burden of Illness study that is available to patients, both adults and children, worldwide. You can read more about the study here: https://www.amihealthy.com/custom/ultragenyx/burdenofillness/burdenofillnesshome.aspx?SITE=XLHNETWORK

Please note that this is your last chance to participate in the Burden of Illness study. December 8, 2014 is the last date for the study to accept information.

Another current opportunity is the "graduates" study by Dr. Whyte, which is open to patients who were treated as children at the Shriner's Hospital in St. Louis. If you're eligible for that study, you can read about it here: http://xlhnetwork.org/files/9613/9931/2887/Graduate_Study_Flyer_Edited2_1.pdf

As always, the XLH Network, Inc. does not counsel individual patients either for or against participation in any specific research study. Prospective volunteers should always carefully review the study's documentation, and discuss the pros and cons of their participation with trusted advisors, including their health care providers and family.

Wednesday, November 12, 2014

Orphan drug status for KRN23 in EU

The treatment for XLH that's currently in clinical trials, KRN23, was recently granted "orphan drug status" in the European Union.

As explained in the press release from Ultragenyx, "The European Commission grants orphan drug status for medicinal products intended to treat diseases or conditions that affect fewer than five in 10,000 people in the European Union. The designation provides certain benefits and incentives in the EU, including protocol assistance, fee reductions, and ten years of market exclusivity once the medicine is on the market." 

Getting this designation is an important step toward bringing KRN23 to the market in the EU. Orphan drug status was already granted in the US in late 2009.

You can read the press release here:


Sunday, November 9, 2014

Midwest XLH Day registration is closed

Registration for our first Midwest XLH Day on November 14-15, 2014 in Indianapolis, Indiana has closed, and confirmation letters for the complimentary hotel rooms are being sent.
If you originally thought you'd be unable to attend, and are now able to, please contact help@xlhday.com to see if it's possible to add you to the registered attendees. You will not be able to register on the day of the event.
For all the registered members, you can see the updated bios of the speakers at http://xlhday.com/?page_id=994 We look forward to seeing you in Indy!

Wednesday, November 5, 2014

Message from the President

The XLH Network is at a watershed moment, where for the first time ever, there's hope for an effective treatment for both children and adults with XLH within the next five years.

The Network's mission involves education, awareness, and support for the XLH community.  Until recently, with no hope for more effective therapies, we've been primarily focused on providing support for our members through the listserv and a website that provides the most current information on XLH, diagnosis, treatment, and ongoing research. 

While our mission remains the same, we have much more work to do today given the explosion of research into bone metabolism and FGF23. It's more important than ever to reach out to everyone diagnosed with XLH and everyone who might diagnose a patient with XLH.

We can't embrace these new challenges without funding. 

2015 must be a year for securing our stability. This year, we raised a fair amount of money from our member donors, the International Society of Pharmaceutical Engineering, and Ultragenyx Pharmaceutical, Inc./Kyowa Hakko Kirin Co., Ltd. Some of the money raised in 2014 was invested in a strategic planning weekend to set goals for our future work, and grant money specifically supported exhibiting XLH information at medical conferences. The remainder of the money was earmarked for two XLH Days (one in Indiana in a few days and another on the west coast in 2015).  

This one year's infusion of money won't last forever, though, and if we want to continue offering our services to an ever-growing membership, we need your help. We work as a virtual organization dependent on hours of volunteer time given by our board. This must change. We need your financial support so we can keep pace with the growing demands of our membership by moving forward with professional staff. A little or a lot, whatever you can contribute would be greatly appreciated. 

Thank you to each one that donated last year. If you have not donated before, please donate this year. Every donation counts. Help us serve adults and children with XLH everywhere. We can't do it without you!


Becky Mock, President

Monday, November 3, 2014

Last chance to register for Midwest XLH Day

Time is running out!

Join us for our first Midwest XLH Day on November 14-15, 2014 at Riley Children’s Hospital in Indianapolis, Indiana! At Midwest XLH Day, you will meet other families dealing with XLH, discuss the challenges, learn how others cope, and develop new strategies for managing your own or your child’s symptoms.

The last day to register for this event is November 8th.

Please go to xlhday.com, learn more about the event, and register now.

We look forward to seeing you in Indy.