News

February 24, 2021February 24, 2021

Study Finds Poor Mineralization in Teeth of XLH Children

News

The teeth of children with X-linked hypophosphatemia (XLH) are marked by defects in mineralization affecting the three layers of hard dental tissue — enamel, dentin and cementum — a small ... Read more

February 18, 2021February 18, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

News, syndicated

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare ... Read more

February 17, 2021February 17, 2021

Trial Will Test Calcitriol on Bone Health; Now Recruiting in Boston

News

A Phase 1 trial is enrolling children and adults with X‐linked hypophosphatemia (XLH) to assess the effects of calcitriol, the active form of vitamin D, on kidney function and bone ... Read more

February 10, 2021February 11, 2021

Pandemic Won’t Stop Rare Disease Day on Feb. 28

News, syndicated

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the ... Read more

February 4, 2021February 4, 2021

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

News, syndicated

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs ... Read more

February 3, 2021February 3, 2021

Case Study Stresses Importance of Early Diagnosis, Awareness

News

The case of a woman diagnosed with X-linked hypophosphatemia (XLH) relatively late in life — after the condition had already taken a substantial toll on her physical health and quality ... Read more

January 27, 2021January 27, 2021

Crysvita Effective at Easing Children’s Pain and Aiding Life Quality, Trial Shows

News

Switching from conventional therapy to Crysvita (burosumab) leads to clinically relevant reductions in pain and fatigue, and better quality of life, in children with X-linked hypophosphatemia (XLH) ages 5 to 12, ... Read more

January 26, 2021January 26, 2021

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

News, syndicated

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to ... Read more

January 21, 2021January 21, 2021

NORD’s Caregiver Respite Program Continues Through Pandemic

News, syndicated

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for ... Read more

January 20, 2021January 20, 2021

Multidisciplinary Approach Key for XLH, Experts Say in Webinar

News

A multidisciplinary approach based on pediatrics, orthopedics, and functional therapy specialties is crucial for managing and treating children and adults living with X-linked hypophosphatemia (XLH), experts emphasized on a recent ... Read more