FDA to consider both pediatric and adult issues

According to a recent Ultragenyx press release, the company has had a meeting with the FDA to discuss the planned submission for approval of KRN23/burosumab, with encouraging results. 

The press release states: "At the meeting, the FDA agreed that the BLA [Biologics License Agreement] can be submitted based on available clinical data and confirmed that both pediatric and adult indications would be included in the review. Based on the agreement, the submission of the burosumab BLA is planned for the second half of 2017."

Of particular importance to the XLH community is the FDA's decision to consider both pediatric and adult indications for the use of KRN23/burosumab, since previously it appeared that the FDA might only consider pediatric use of the new therapy during its review under the Breakthrough Therapy designation. Without FDA approval for adults, insurance companies might only cover the treatment for children, not for adults. Anecdotal evidence suggests that the treatment can be extremely beneficial for adults, not just for children.

The Network is investigating a variety of ways to provide the FDA with information from the patient perspective, particularly with respect to adult issues that need treatment, which have not been addressed as widely in the medical literature as the pediatric issues. We'll let you know  how you can help too as soon as we have more information.

You can read the entire press release here: 
http://ir.ultragenyx.com/releasedetail.cfm?ReleaseID=1031181

Weak Bones, Strong Wills!

Our book of the XLH community's experiences with the condition, Weak Bones, Strong Wills, the Stories of XLH has been compiled and edited, and now is going through a final proofreading. We think you'll find the stories inspiring, and you'll recognize many of the experiences from your own life or a family member's life.

We're on schedule to have paper copies available for purchase and autographing during XLH Day, and both digital and paper copies will be available then from online retailers like Amazon.com, BN.com, and Kobo.com. We'll share the links as soon as they're available.

Meanwhile, you can see a rough mock-up of the cover art (still a work in progress) at our official facebook page, https://www.facebook.com/xlhnetwork/?ref=bookmarks

Thanks to everyone who submitted a story!

McGill Video

McGill University held a symposium last year on "Soft Bone and Tooth Diseases -- what can we do about them?"

You can watch the video of the symposium here: http://www.canalsavoir.tv/videos_sur_demande/cafes_IRSCCIHR_2016/softbones_toothdiseases

Dr. Frank Rauch reviews the basics of XLH, starting at about 10 minute point. Then, at about 21 minutes, Dr. Marc McKee talks about the mineralization process in teeth and the problems that XLH causes for mineralization.

As an added benefit, if you speak French (or at least learned a bit of it), you can follow along with the French subtitles!

XLH Day registration to begin July 9

It's almost time to register for XLH Day, which will be held on October 6-7 at Quinnipiac University's Frank H Netter MD School of Medicine in North Haven, Connecticut. We expect the registration site toopen July 9th and close September 16. Please don't try to register before we announce it's open; early registrations will be deleted during testing.

Meanwhile, we're sharing the graphic for this year's event, which you can see at our official facebook page, facebook.com/xlhnetwork. Make sure you're following us there, so you get the most recent news and information.

The graphic merges aspects of the XLH disease process with a Connecticut legend. Hidden in plain sight as the trunk of a Charter Oak tree are leg bones with the varus deformity common to many XLHers.

The legend of the Charter Oak started when King Charles II granted the Connecticut Colony its charter in 1662. His successor consolidated the New England colonies, and when the royally appointed Governor came to collect the charter, heated argument ensued, the candles within the room were blown out, and the charter was spirited away. The charter was hidden in Hartford, in a white oak tree henceforth known as the Charter Oak. Today, you can find the symbol of the Charter Oak on the back of the Connecticut state quarter.