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Health & Fitness

Shriners Docs Find New Hope for Kids with Rare Bone Disease

Researchers at Shriners Hospitals for Children in Frontenac publish new treatment for rare bone disease in New England Journal of Medicine.

It's been an exciting day here at Shriners Hospitals for Children in Frontenac. Today, our own Dr. Michael P. Whyte has a study published in the New England Journal of Medicine. Dr. Whyte is the medical/scientific director of our hospital's Center for Metabolic Bone Disease and Molecular Research - also known as the Research Center - that investigates, treats and diagnoses more than 100 rare bone diseases.

News of the article and the findings were popping up major news websites throughout the nation, making us even more proud of all the talented folks in our Research Center.

Just last week, I posted some observations from the staff in that department in celebration of Rare Disease Day. And I have to say it again, I think the residents of Frontenac should be extremely proud that some of the leading medical professionals in orthopaedics and rare bone diseases are doing their work right here in your own backyard.

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The study published in today's New England Journal of Medicine is pretty fascinating and incredibly hopeful for children born with this rare and sometimes life-threatening disease, hypophosphatasia, a condition that affects bone metabolism, blocking important minerals from depositing in the skeleton. Basically, that means kids can experience anything from losing teeth too early, to bone weakness (rickets), and even respiratory failure and death. Only about 1 in 100,000 children are born with it.

The study shows that our doctors may have identified a promising new treatment for the disease, which, until now, had been untreatable.

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I'll attach the full article from our website, but what I think is incredible is that after just six months of the treatment, most of the patients saw substantial healing of rickets. After a year, children who needed respiratory help were breathing unaided. All of the children in the study who completed one year made progress in sitting, crawling, pulling to standing, and even taking steps. 

It's amazing work - and we're so proud that it happened right here at Shriners Hospitals for Children - St. Louis. I hope you'll take a couple of minutes to read the full story on our website and take a look at the Xrays from one of the patients in the study.

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