“To be only six years old and have seven breaks and two surgeries, that’s a lot,” Jessica said. “Some days are really hard.” Still, Everlee refuses to slow down.

Baylor College of Medicine has received new funding from the National Institutes of Health for two consortia in the Rare Diseases Clinical Research Network: $7.4 million over five years to renew the ...

A Queen’s-founded startup is translating long-term research into potential treatments for rare genetic bone disorders.

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week. Currently, ...

A new biopharmaceutical company is devoted to developing therapies for rare bone diseases in Montreal. Ocythera was launched earlier this month by adMare BioInnovations, one of Quebec’s largest ...

A San Francisco boy with a rare bone marrow disease has been on the waitlist for more than a year. One of his biggest challenges is finding a donor that matches his specific ethnicity: Chinese and ...

On Sunday, Sept. 17, attorney and former Brooklyn Republican Party Chairman Craig Eaton and his wife Susan hosted “Funtasia,” an annual event geared to raise money for their Multiple Hereditary ...

This week's Kid Captain for when the Hawkeyes take on the Ducks is 10-year-old Harper Atkinson of Wapello. Atkinson has a rare bone disease. In fact she may be the only person in world with her ...

(RTTNews) - Mereo BioPharma Group plc (MREO), a clinical-stage biopharmaceutical company, provided an update on its lead programs for its rare-disease product candidates, setrusumab and alvelestat, ...

In a recent study, Associate Professor Makoto Ikeya (Department of Clinical Application) and his team of researchers successfully established a method to generate jawbone-like organoids (mini-organs) ...

Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like ...

When Casey McPherson became a father, his purpose became clear. He needed to raise his daughter, Rose, to be loving, courageous, healthy and strong. He needed to protect her. Many parents can ...