A fusion protein that ferries a healthy version of a bone-related enzyme gone awry has shown early clinical success in treating a rare bone disorder with no known therapy, researchers reported earlier this month at the Endocrine Society's linkurl:annual meeting;http://www.endo-society.org/endo/ in Washington, DC. The drug -- which is essentially a protein-based enzyme delivery mechanism -- could open the door to treatments of other skeletal disorders that have so far been deemed untreatable.
"This is probably the most promising approach so far" for metabolic bone disorder therapies, linkurl:Sundeep Khosla,;http://mayoresearch.mayo.edu/staff/khosla_s.cfm an endocrinologist at the Mayo Clinic in Rochester, Minn., who was not affiliated with the work, told __The Scientist__. The new therapy targets a rickets-like disease called linkurl:hypophosphatasia;http://ghr.nlm.nih.gov/condition=hypophosphatasia (HPP), which is caused by a single mutation in the gene coding for an enzyme known as tissue non-specific alkaline phosphatase, or TNSALP....
and adult (right) hypophosphatasia
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