Inherited Diseases

Research hospital announces blindness treatment, new leadership

Published in Biotech Business Week, January 10th, 2005

The development of a laboratory model for a rare, inherited form of blindness holds promise that scientists might one day be able to test new treatments to prevent or cure this devastating disease of the retina.

This finding, from investigators at St. Jude Children's Research Hospital and Columbia University, was published in Molecular Brain Research (MBR).

The model for this disease, called Leber congenital amaurosis (LCA), is especially important because no treatments are currently available to prevent it. There are not enough patients to enroll in large clinical trials to test new prevention treatments; therefore, any potential new therapy...

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Source: Biotech Business Week (2005-01-10)

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