Wednesday, September 5, 2018

Burosumab approved for pediatric use in UK

We are absolutely thrilled to announce this morning that burosumab has been APPROVED for routine use on the UK’s NHS for children from 1 years old up to young adults aged 16/17 years.
This news completely changes the landscape for the estimated 250 children living with XLH in the UK. Burosumab will be the first and only treatment that targets the cause of the hypophosphatemia in the body. The treatment helps reduce symptoms during childhood and by reducing bone damage during childhood growth it could mean the potential for a better quality of life for children later in their lives too.
We want to say a MASSIVE thank you to all those individuals who shared their experiences with us and with NICE so that decision-makers could understand what this treatment will really mean to patients and their families. Without all of your input this decision to approve burosumab may not have been made.
For those that can’t quite believe their eyes this morning, please visit NICE for the full guidance on children with XLH. If you have further questions about the treatment and whether it is right for your child then please do speak to their paediatrician that manages their XLH.
Since this news is so important, we at XLH UK have put together a press release. See link.
https://www.dropbox.com/s/o97z8m8nswd79m2/PR_xlhuk.pdf?dl=0
Whilst access to burosumab on the NHS will just be for children for now, let me assure you that the fight for the adult license has begun.
Meanwhile please please do stay tuned and encourage other XLHers in the UK & their families to join our XLH-UK facebook page because we’re so much stronger together.

Friday, August 17, 2018

Pain Comments to FDA

The FDA recently hosted a Patient Focused Drug Development meeting on Chronic Pain, and the public is invited to provide comments about the information presented, as well as their own experiences with pain. The XLH Network, Inc., provided a full statement on behalf of our community, and a small portion of that statement is included below. We want to encourage our members to review the PFDD information at https://www.fda.gov/Drugs/NewsEvents/ucm603093.htm and to write their own responses and comments if they believe the information will help the XLH community. You can submit your comments here: https://www.regulations.gov/document?D=FDA-2018-N-1621-0001. Comments are due by September 10th.
Pain is a life-long part of the XLH experience that includes chronic bone pain (either due to fractures or due to mineralization defects without fractures), osteoarthritis at an earlier age than the general population due to cartilage thinning, dental pain from spontaneous abscesses, neurological pain from calcifications that impinge on the spinal canal, and pervasive, early-onset bone spurs (enthesophytes) that affect many of the tendon and ligament insertion sites throughout the skeleton and spine.
The most significant downsides to the available current treatments for the pain of individuals with XLH are as follows: 1) they not effective in some patients; 2) they work somewhat but cause significant adverse effects from the drug; 3) they work but at the risk of addiction or stomach, kidney, or liver damage; 4) generally they don't completely resolve the pain, just dull it a bit; and 5) they are a treatment and not a cure, because they don't get to the root cause of the pain.
Some individuals with XLH report having difficulty getting effective dosages or sufficient quantities of pharmacological treatments to handle the extreme severity of pain they experience. Other patients report being viewed as drug-seeking or having a psychosomatic complaint because their pain is both diffuse and severe. In addition, the origin of pain often evolves from time to time without an obvious clinical cause to explain the source of the pain. Finally, many health care providers are unaware that XLH is associated with pain and/or are unaware that there is such a thing as bone pain in the absence of trauma, and this may result, too often, in accusations of drug-seeking behavior or as having psychological issues.

Monday, July 30, 2018

Second virtual walk/run

We are kicking off our 2nd annual Virtual Walk/Run for THE XLH NETWORK, INC as a way to countdown to Baltimore! Our virtual walk/run will take place from July 30–October 7th.
Spread the word and raise awareness by creating a peer to peer page and encouraging your friends, family members, colleagues and others to support your efforts. By becoming a fundraiser for THE XLH NETWORK INC, you will help us continue our mission to help children and adults living with XLH. Raising funds for the Network helps us continue to advocate for you and your families.
For more information and to sign up:https://xlhnetwork.networkforgood.com/projects/54586-2nd-annual-walk-run-for-xlh

Wednesday, July 4, 2018

Last chance to comment on UK decision about burosumab

There are only a few days left to speak up about the benefits of burosumab before the regulatory agency makes its final decision, possibly denying this life-changing treatment to patients in the UK. To comment, you must register at NICE using this link
The evaluation committee is interested in receiving comments on the following:

a)How do symptoms (both physical and psychological) and treatments (including any surgery) you or your child experienced in childhood affect you in adult life?
b) For children on standard treatment (phosphate and calcitriol), what are the side effects of taking current treatment (phosphate, calcitriol)? How is that a burden for the child and your family?
c) If you have a child who is 1-12 years old and on burosumab, please explain how this treatment is currently helping in the short-term and how you expect that it will help in the long-term.
d) If you have a child who is 13-17 years old and on burosumab, please explain how this treatment is currently helping in the short-term and how you expect that it will help in the long-term.
If you have any questions at all, please don't hesitate to get in touch. Comment below or email us at Oliver.gardiner@xlhnetwork.org

Tuesday, July 3, 2018

Survey on adults living with familial hypophosphatemia

The Pre-Event survey for the Symposium will help us to have even a bigger impact on our audience members, which include the medical community, researchers and insurers. Make sure your voice is heard! It will only take a few minutes. Click here to start:

https://docs.google.com/forms/d/e/1FAIpQLSe-dwpdUVrZ7-GauuFK3xDBjIRYUPiYr1s4QJe1v-cyCOMzMQ/viewform

Wednesday, June 27, 2018

Executive Director, Rachael Jones

We are excited to announce that Rachael Jones, a fellow XLH Network member, has agreed to assume the position of Interim Executive Director of the XLH Network effective with the resignation of Carol Lafleur at the end of this month. Rachael brings a wealth of nonprofit experience to this interim position, and as an XLH patient herself, she brings firsthand knowledge of what it means to live with XLH.
Rachael was born in Colorado and recently moved back to Colorado after spending most of her life in various states in the Southwest region. Rachael is married and has two young children. She spent several years as an administrator for non-profit organizations. She also loves to write and spent a couple years editing and writing curriculum for elementary students.
Rachael is passionate about the work that The XLH Network, Inc. does for XLH patients and caregivers. After attending XLH Day in 2013, she became involved in subsequent XLH Days to help with children’s programs. The daughter of a spontaneous case, Rachael was diagnosed at birth with XLH, and her brother, two nieces, and her own two children also have the condition. In all, there are seven people in her family who have XLH. Rachael desires to find more ways to connect XLH patients and families, and as part of her passion for children and writing, she plans to help develop materials for children with XLH.
Please join us in welcoming Rachael!
The XLH Network, Inc., Board of Directors

Monday, June 18, 2018

Help ensure access to life-saving treatment!

The regulating authority in England is set to decide, incorrectly we believe, that even though burosumab is safe and effective, it will not be made available to patients in the UK for financial reasons. There is a short window of opportunity (just until July 6) for patients' voices to be heard (from anywhere in the world, but especially from the UK), so speak up now! And share this post and ask your friends in the XLH community to act too!
To comment, you must register at NICE using this link.
The evaluation committee is interested in receiving comments on the following:
  • How do symptoms (both physical and psychological) and treatments (including any surgery) you or your child experienced in childhood affect you or your child in adult life? 
  • For children on standard treatment (phosphate and calcitriol), what are the side effects of taking current treatment (phosphate, calcitriol)? How is that a burden for the child and your family?
  • If you have a child who is 1-12 years old and on burosumab, please explain how this treatment is currently helping in the short-term and how you expect that it will help in the long-term.
  • If you have a child who is 13-17 years old and on burosumab, please explain how this treatment is currently helping in the short-term and how you expect that it will help in the long-term.

We believe in the power of our combined voices, and there is a reason NICE provides a window for discussion. It is imperative that we stand up for children and families in the UK and that we do so now. Please join us.
Finally, if you have any questions, feel free to email: oliver.gardiner@xlhnetwork.org