Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.
Treatment with an antibody targeting a protein called sclerostin improved bone mass and strength in a mouse model of X-linked hypophosphatemia (XLH), a study shows. The study, “Sclerostin antibody treatment increases bone mass and normalizes circulating phosphate levels in growing Hyp mice,” was published in the Journal…
The prevalence of X-linked hypophosphataemia (XLH) rose in both children and adults across the United Kingdom between 1995 and 2016, a study found, likely due to improvements in clinical testing. Mortality rates were higher in elderly people with XLH than in people of similar age without this disorder serving…
More than 80% of people with X-linked hypophosphatemia (XLH) develop hyperparathyroidism as a side effect of treatment with oral phosphate and active vitamin D, a study reports. Tertiary hyperparathyroidism, which results from prolonged hyperparathyroidism, is hard to treat. Surgical removal of the parathyroid glands is not…
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…
Extended use of Crysvita (burosumab) for up to 48 weeks maintains normal blood levels of phosphate, relieves pain and stiffness, and aids the healing of bone fractures and overall physical condition of adults with X-linked hypophosphatemia (XLH), an extension study of a Phase 3 trial shows.
The U.S. Food and Drug Administration (FDA) approved a label update for Crysvita (burosumab) to cover treatment of X-linked hypophosphatemia (XLH) for patients 6 months and older. This update includes data showing improvement in stiffness, continued healing of fractures, and maintenance of efficacy with longer-term use of Crysvita…
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