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Author Topic: For journalists  (Read 2878 times)

GinJones

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For journalists
« on: January 12, 2016, 09:52:38 PM »
If you're a journalist looking for information about XLH, we recommend:

1. Our website, xlhnetwork.org

2. Our facebook page, facebook.com/xlhnetwork

3. Our youtube channel: youtube.com/xlhnetworkincvideo

4. Our book, Weak Bones, Strong Wills, The Stories of XLH, available at all major online distributors, including Amazon: https://www.amazon.com/dp/B075MQMZXG/

5. The National Organization for Rare Diseases (NORD) page on familial hypophosphatemia, which is another name for XLH: http://rarediseases.org/rare-diseases/familial-hypophosphatemia/

6. For information about genetic transmission of dominant disorders: http://ghr.nlm.nih.gov/handbook/inheritance/inheritancepatterns

7. An overview of the condition, its causes, diagnosis and current standard of care (before burosumab), written by several of the top U.S. experts in XLH, and published in the Journal of Bone and Mineral Research, "A Clinician's Guide to X-Linked Hypophosphatemia" is here: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3157040/


If you would like to speak with a representative of the organization:

Executive Director: ExecutiveDirector@xlhnetwork.org
President: president@xlhnetwork.org
Treasurer: treasurer@xlhnetwork.org
« Last Edit: February 17, 2018, 09:30:31 PM by GinJones »

GinJones

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Re: For journalists
« Reply #1 on: November 12, 2017, 05:43:20 PM »
2018 has the potential to be a life-changing year for the XLH community. 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 every year since 2001, 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.

« Last Edit: January 16, 2018, 10:16:59 PM by GinJones »

GinJones

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Re: For journalists
« Reply #2 on: December 15, 2017, 05:11:49 PM »
Excellent news for the European members of our community! According to a press release today from Ultragenyx and Kyowa Hakko Kirin, "the Committee for Medicinal Products for Human Use (CHMP), the European Medicines Agency's (EMA) scientific committee, has adopted a Positive Opinion recommending the conditional marketing authorization of burosumab, an anti-FGF23 human monoclonal antibody, for the treatment of X-linked hypophosphatemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons."

There's more work to be done before the treatment will be available to patients in Europe, but this is a huge leap forward! A final decision needs to be made by the European Commission, and is expected in the first quarter of 2018. It will apply to all 28 countries of the European Union, Norway, Iceland and Liechtenstein, but individual countries will still need to do additional reviews, largely with respect to payment issues, rather than safety and efficacy issues.

You can read the entire press release here: http://ir.ultragenyx.com/releasedetail.cfm?ReleaseID=1051923
« Last Edit: December 15, 2017, 05:17:13 PM by GinJones »

GinJones

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Re: For journalists
« Reply #3 on: September 06, 2018, 05:05:35 PM »
The XLH Network, Inc.
Yesterday at 4:04 AM ·
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 XLH’ers in the UK & their families to join our XLH-UK facebook page because we’re so much stronger together.

https://www.nice.org.uk/…/gid-hst1…/documents/html-content-2