Message from the President, Bill Coogan

It's not often that something life-changing happens to an entire global community, but that could be true for XLHers of all ages in 2018. Next year may see the approval, all around the world, of the first-ever treatment for XLH (and the related hypophosphatemias) that gets at the root of the problem (phosphate wasting).

But the treatment will only help if the relevant people know about it! Our next big challenge will be to reach everyone in both the patient population and the medical community and then to educate every last one of them about the realities of living with XLH, why treatment is necessary and what the treatment options are.

At the moment, there's a huge disconnect between what the experts know and what's happening in the day-to-day treatment of XLHers by non-experts. While some medical providers are current on their understanding of XLH, there are far too many instances of uninformed medical providers telling patients that there's nothing that can be done to help them (or that their symptoms are unrelated to XLH). Some patients can challenge the bad advice, but may not be able to find any better medical provider, depending on where they live. Or they may lack the knowledge or the resources to successfully challenge their medical providers' statements.

We plan to work on both sides of those conversations. Some initiatives will focus on educating the medical providers so they'll give better advice, and other projects will focus more on educating the patient community about current options so no one will be discouraged by outdated advice.

For the health care providers side of the equation, we'll be attending more medical conferences than ever, including possible going to the meetings of the American Association of Nurse Practitioners and the American Academy of Clinical Endocrinologists; and holding a Patient-Focused Drug Development meeting (attended by a representative of the FDA) in conjunction with XLH Day 2018 (in the Baltimore/Washington area) to create materials (patient testimony and guided discussions that are videotaped and then transcribed for dissemination) about the adult symptoms of XLH.

For the patient side of the equation, we'll be creating and sharing some short videos by experts on various aspects of XLH and publishing age-appropriate materials to help children understand their XLH. Plus, as we've done for the past seven years, we'll be offering the community all the resources of XLH Day, with expert speakers and a chance to network with other XLHers and their families.

But first, we need to be sure we have the resources we need to carry out these initiatives at a critical time for the XLH community. In a few weeks, we'll be participating in #GivingTuesday (November 28th), and before then we'll share the details of how you can get involved, not just by sending money, but also by sharing our Tweets and Facebook posts in your social media circles.

You don't have to wait until then, of course! We aren't waiting to start the necessary work, after all. Donations can be made here any time: https://xlhnetwork.networkforgood.com/projects/29168-every-day-giving

Looking forward to the next twenty years

Message from the Network's President, Bill Coogan:

We've already reviewed some of the milestones of the past twenty years, and now I'm here to share some of our expectations for the milestones of the coming year and beyond. If you want to see these goals come to fruition, we need your financial support now. You can donate here: https://donatenow.networkforgood.org/xlhnetwork?code=donate%20page

Already, 2017 is shaping up to be an eventful year. We hope to announce the hiring of an Executive Director soon, who will help us to accomplish our more substantive goals. 

In addition to our regular activities that we've engaged in for the past twenty years, of representing, supporting and advocating for the XLH community (and those with related phosphate-wasting disorders), we'll be particularly involved in the following major programs:

• Organizing the next XLH Day to be held at Quinnipiac University's Frank H. Netter School of Medicine (in North Haven, Connecticut) on October 6-7, 2017. 
• Collecting data for a Natural History of XLH (and related disorders).
• Publishing educational materials geared toward children, providing age-appropriate information about XLH (and related disorders).
• Publishing Weak Bones, Strong Wills, The Stories of XLH, a collection of essays about the XLH experience. 
• Representing the XLH community (and those with related phosphate-wasting disorders) as KRN23 goes through the regulatory process. 

I'm sure you'll agree that these are all critical projects that need to be undertaken sooner rather than later, and none of them are without cost. If we're to do everything on our agenda, we need your financial support. 

Please help us to help you. Donate now: https://donatenow.networkforgood.org/xlhnetwork?code=donate%20page

Thank you.

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.

Message from President Bill Coogan

It is an exciting time to serve as interim president of the XLH Network. Thanks to the leadership of my predecessor and the many contributions of the board of directors, the scientific advisory board, and our other generous volunteers and donors, we have numerous projects in the works.

Last year we hired administrative assistant, Stacy Duck, and we're now beginning the process of hiring an Executive Director during the second half of this year. Stacy has already completed a number of administrative projects that we'd never had time to do before, and we expect that hiring an Executive Director will enable us to expand by leaps and bounds the amount of work the volunteer officers and board can accomplish on behalf of our members.

The board and I will also be representing XLH patients and their families at numerous venues this year. We will be at the Endocrine Society conference in Boston on April 1-3, the American Society for Bone and Mineral Research conference in Atlanta on September 16-19, along with an assortment of other patient-advocacy events in the U.S., the U.K. and Europe. We're hoping to arrange a time to meet local XLHers when we're at those events, so if you're in the area and would like to say hello, please email me at bill.coogan@xlhnetwork.org.

Last, but not least, we are hard at work planning XLH Day, to be held at the Shriners Hospital for Children in Houston, Texas, on August 12-13, 2016. We'll have livestreaming for those who can't attend in person, and afterwards there will be videos of the main presentations available at our new Youtube channel, https://www.youtube.com/channel/UCOCxS6CV6NeNxoFFivOyNpg.

Please keep in mind that even though we have been fortunate in obtaining grants for our two new employees due to the generosity of Ultragenyx Pharmaceutical, those monies do not cover the rest of our budget, like the cost of our attendance at medical conferences or the expense of this year's XLH Day. If you'd like to make a donation, the information you'll need is here: http://xlhnetwork.org/index.php/donate/

I look forward to updating you on our activities as the year progresses, and I thank you for your support.

Message from Network President Becky Mock

It has been an honor and a privilege to serve as the President of the XLH Network for the past three years, but it is time for me to pass the gavel to someone else. I will be stepping down as president at the end of this year, although I will continue to work as an at-large Board member.

Current Vice-President, Bill Coogan, will be stepping up to the presidency. Many of you have met Bill and know, as I do, that he brings a great deal of dedication, skill, and personality to the organization.

Over the past few years, we've accomplished a great deal—the Board of Directors, the membership, and I, all working together. It has been my mission to transition the Network from an all-volunteer group to one with paid staff, and I am pleased to say that we have taken the first steps toward that goal.

During my tenure, two grants were funded by Ultragenyx Pharmaceutical, one to underwrite a substantial portion of the cost of hiring an administrative assistant, and the other to do the same to hire an executive director. The administrative assistant is in place. The process for hiring the executive director has begun, and I am confident that with grant funds available and the recruitment process underway, we will move forward and hire our first executive director in 2016.

I am proud that we have seen XLH Days become life-changing experiences, offering both educational opportunities with expert presentations about XLH, and fun times helping people with XLH meet "someone like me," perhaps for the first time ever. We know how important it is to bring XLH Day to all parts of the country. We’ve held events in New York, Connecticut, Indiana, and California. Work is well underway for the Texas XLH Day in 2016. We’ll get back with more information and a definite date as soon as we can get the logistics sorted out.

We cannot take a rest. We must forge on. The XLH Network is the ONLY organization that advocates for you and others with XLH. There is much more to do to maintain our new initiatives and advance our future projects. We cannot do all that is needed with the funds we have now. Our budget is tight, focused and designated for the work we're doing today. To maintain, we need your help. To grow, we need more donations and support. We are the best investment you can make if you wish to make a difference in the lives of everyone with XLH.

Finally, I would like to thank everyone that has volunteered or made a donation during my presidency. I'm counting on you to offer that same support to future presidents. Please help the Network continue to advocate for everyone with XLH and their families.

We can't do it without you!

Sincerely,

Becky Mock, President

Message from the Network President

From Network President Becky Mock: 

What an amazing year for the XLH Network! We’ve accomplished so much in 2015, thanks to your support. We've hired our first-ever employee, an administrative assistant who will assist with the daily operations, allowing Board members and volunteers to do more for Network members. We've established a template for XLH Days, which will make it possible to hold events in different locations around the United States.

Other critically important efforts included participation in patient advocacy and medical research conferences, where we met with numerous patient advocates and activists with the unified goal of educating, networking, and sharing best practices. We have been instrumental in assisting with recruiting patients for clinical trials this year and with distributing the Ultragenyx Burden of Illness Survey documenting the pain and suffering people with XLH endure every day, which has not been done at this level before. (It’s still open. Add your experience to the survey if you haven’t already: https://www.amihealthy.com/custom/ultragenyx/burdenofillness/burdenofillnesshome.aspx?SITE=XLHNETWORK.)

We are at a critical time in the growth of the XLH Network. We must be present and visible in front of leaders of the orphan drug and disease community: National Institutes of Health, pharmaceutical industry, academia, and the FDA. Current clinical research trials for children and adults may hold the key to a promising treatment. Negotiating the lengthy FDA approval process and bringing a new treatment to patients are complicated activities and take persistence. Only at national events such as those listed above is everyone brought together to discuss the best methods of researching, diagnosing and treating rare diseases in the 56 countries in which we know our members live.

We must educate more medical professionals, especially pediatricians and dentists, about the symptoms of XLH. We need to find more ways for members to meet. And finally, we need to do more as patient advocates, raising our voices so patient centered outcome studies will be a mandatory part of all work done by researchers, clinicians, and the FDA. We must work together to make this happen.

As 2015 comes to an end, please consider making a donation to our year-end campaign: http://xlhnetwork.org/index.php/donate/

Please help the XLH Network continue to work on behalf of everyone with XLH, their children, and their families. We can't do it without your help. Thank you!

President's message

The Network’s Board of Directors held its annual in-person meeting the first weekend of August this year, and while words are inadequate to convey the degree of dedication, excitement and anticipation in the air, I can give you a taste of the projects we’re currently working on or have planned for the rest of the year.

One of the biggest topics of discussion was the future of XLH Days. The last two (MidWest XLH Day and West Coast XLH Day) were great experiences, and we are looking forward to meeting more of our members at future events. The board formalized its intent to hold one XLH Day each year, generally in the summertime. While there are many, many details still to be worked out (and both volunteers and financing to secure), based on the enthusiasm shown by members in Texas recently, we have begun looking into holding the next XLH Day in the vicinity of Houston in late July or early August of 2016.

We also created an ambitious calendar for the board. This fall, we will represent our members at the American Society for Bone and Mineral Research (ASBMR) conference in Seattle and the National Organization for Rare Disorders (NORD) event in DC. We’ll also be attending workshops on how best to interact with the medical research community in the context of the Patient Centered Outcome Research Institute (PCORI.org). For the future (2016 and beyond), we’re looking into the costs and benefits of eventually expanding our representation to conferences of pediatricians, dentists, and orthopedic surgeons.

A great deal of time was spent on some major projects that are either in early, confidential stages or are a bit further along, but not quite ready to be unveiled. You’ll hear more about them over the next six months, and I’m certain you will be as impressed as I am by the dedication of your Board members in pursuing these time-consuming but incredibly valuable initiatives. Based on our hard but often invisible work over the last two or three years, the XLH Network, Inc. is positioned for significant growth in the coming year.

Last, but not least, we spent a substantial amount of time reviewing our financial condition and figuring out how we are going to pay for our existing programs as well as the ambitious new initiatives. We’re confident, however, that our members will step up to ensure that each XLH Day will be even better than the one before, that we can continue to advocate on your behalf, and that XLHers will always be able to turn to the Network to support you through the challenges of living with XLH or caring for someone with the condition. In that vein, it would be remiss of me not to mention that information on donating to the Network can be found here: http://xlhnetwork.org/index.php/donate/

I look forward to moving ahead with the plans made at this meeting and sharing the details of the new initiatives as they are finalized.

Becky Mock
President, XLH Network, Inc.

Message from the President

The Network's Board met on Sunday to select the portions of our Strategic Plan that we intend to accomplish this year.  Here’s a bit on the priorities for 2015.

Plans are underway for West Coast XLH Day to be held on April 9-10, 2015 in Novato, California.  Watch for more details to be posted soon on our website. Ultragenyx, the pharmaceutical company leading the development of KRN23, is hosting a Patient Day on Saturday, April 11, also in Novato. Thirty travel scholarships are available for people interested in attending.  Our events coincide, so consider applying for a travel scholarship to attend both.  Ultragenyx invited other rare disease organizations, so if you are interested, please apply today: http://events.r20.constantcontact.com/register/event?oeidk=a07ea1v5hyo78a40bff&llr=orxhodsab

We are joining with other rare disease groups for Give Rare Day on March 3 to promote the fact that more people are affected by a rare disease than by cancer and AIDs combined.

Our social media will increase with the creation of a YouTube channel and a way to share photographs. An upcoming website upgrade will make it easier for members to find information and support.

Our important advocacy and education efforts will continue. The XLH Network will represent our members at ASBMR (American Society for Bone and Mineral Research) and other conferences to strengthen connections to researchers and doctors.

We hope to hear from you in 2015, either in person at the West Coast XLH Day or through our virtual platforms.  Working together, we do make a difference!

Becky Mock

President, XLH Network, Inc.

Thank you!

Message from the XLH Network's President:

This has been a tremendous year of growth. We've made incredible advances in educating members, doctors, and the medical community about XLH and its impact on our everyday life. It’s an exciting time for everyone with XLH, especially with a new treatment in development for the future.

Thank you for your dedication and support of the XLH Network. There is still time to make your year end DONATION and support work for a better future for ourselves and future generations. You can help us continue to make a difference in the lives of people with XLH.

The Board of the XLH Network, Inc. is excited that 2015 has the potential to bring real change, and we hope you are as well. Thank you for your generosity and investment in the work we do on behalf of families with XLH.

 Becky Mock, President

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!

Sincerely,

Becky Mock, President

President's Review of 2013

Network President, Becky Mock, is here to remind us of last year's accomplishments: 

The fall of 2013 was a particularly exciting and busy season for the XLH Network, Inc.

In mid-September, we teamed up with seven other rare bone disease groups to form the Rare Bone Disease Advocacy Alliance to meet with members of Congress to discuss the challenges of life with a rare bone disease and to advocate for greater public awareness and increased federal funding for research. I met with some of the North Carolina caucus, including Representatives David Price and Robert Pittenger, and the Legislative Assistant from Senator Kay Hagan's office.

The following weekend was XLH Day, held at Quinnipiac University in North Haven, Connecticut. People traveled from all over North America, as far as Texas, California and Canada, to meet fellow XLHers and hear from medical providers with substantial experience treating XLH patients. If you weren't able to be there in person, you can still benefit from the lectures, which were videotaped and are available at http://www.youtube.com/watch?v=IIAtcMGaUVU and http://www.youtube.com/watch?v=ay8LdZspmco

Finally, in October, the majority of the Board was able to gather at the annual meeting of the American Society for Bone and Mineral Research in Baltimore, Maryland to network and discuss XLH with the leading experts in bone disease.. You may have already seen the news, that a member of our Scientific Advisory Board, Thomas Carpenter, M.D., won the 2013 Most Outstanding Clinical Abstract Award at this event.

We're working hard to make this year even more productive. I'll be back in a few weeks to share our plans for 2014.

2014 Officers, Directors

From Becky Mock, President of the XLH Network, Inc.:

On behalf of the entire Board of Directors, I'm pleased to announce that the following slate of officers has been elected for 2014:

• Becky Mock, President
• Bill Coogan, First Vice President
• Geoff Edelson, Treasurer
• Joyce Inman, Secretary

Board members that continue to serve terms on the Board include Chris Younger, Kathy Buchanan and Oliver Gardiner.

I'm very pleased to announce that Gin Jones has joined the Board for a two-year term, from January 1, 2014 through December 31, 2015. Gin is a lawyer, writer and XLH patient. In her spare time, she writes mystery novels, grows garlic and makes quilts. 

The work of our volunteers is demanding and time consuming. I appreciate the dedication and commitment of each Board member. Without our volunteers, we would not have a Network. Thank you!