Stargardt's Disease - Moving to Clinical Trials

On Friday, April 6, 2007, a symposium was held at the Coleman Center in New York under the auspices of the Foundation Fighting Blindness. The symposium was co-chaired by Gerald A. Fishman, M.D., and Rando Allikmets, Ph.D. A list of participants and agenda is included at the end of the document.

The intended purpose of the meeting was to address issues relevant to moving pre-clinical studies of Stargardt disease into clinical trials.

Introduction
Stargardt disease, initially described by the German ophthalmologist Karl Stargardt in 1909, is predominantly an autosomal recessively inherited disorder, although an autosomal dominant Stargardt-like phenotype has also been described. The estimated frequency of the recessive form is 1 out of 8,000 to 10,000 in the United States. The dominant form of dystrophy is approximately ten times less frequent than the recessive form. For the purpose of this document, the disease described in 1963 as fundus flavimaculatus, by the Swiss ophthalmologist, Adolphe Franceschetti, and recessive Stargardt disease are considered as synonymous. Mutations in the ABCA4 gene have been identified in the recessive form of Stargardt disease. The dominant form of Stargardt-like dystrophy is associated with mutation in a different gene, ELOV4.