New therapies for skeletal dysplasia

Project Details


Inherited disorders of the skeleton are common with significant health impacts. My research has enabled treatment of these conditions at their genetic source. This program will assess whether drug treatment of infants with achondroplasia will decrease the 50-fold higher risk of sudden death by relieving pressure on the spinal cord, and assess whether treatment of children with metaphyseal dysplasia will relieve pain, improve skeletal deformity and eliminate the need for invasive surgery.

Effective start/end date1/01/18 → …


  • United States-Israel Binational Science Foundation (BSF)


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