New therapies for skeletal dysplasia

Project Details

Description

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.

StatusActive
Effective start/end date1/01/18 → …

Funding

  • United States-Israel Binational Science Foundation (BSF)

Fingerprint

Explore the research topics touched on by this project. These labels are generated based on the underlying awards/grants. Together they form a unique fingerprint.