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LRAT

Disease Category: autosomal recessive

Patient Population:

Known Clinical Trials: 

Research Studies: 

Treatment Options: 

Strategies to Preserve Eye Health: 

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Image by Jordan Cox

Jane Doe

France

A FACE OF RP

Long-Term Follow-Up of Retinal Degenerations Associated With LRAT Mutations and Their Comparability to Phenotypes Associated With RPE65 Mutations

Mays Talib, Mary J van Schooneveld, Roos J. G. van Duuren, Caroline Van Cauwenbergh,  Jacoline B. Ten Brink, Elfride De Baere, Ralph J Florijn, Nicoline E Schalij-Delfos, Bart P. Leroy, Arthur A. Bergen, Camiel J. F. Boon, 

Translational Vision Science & Technology, Vol 8, 24, 19 Aug 2019. doi:10.1167/tvst.8.4.24

Purpose: To investigate the natural history in patients with LRAT-associated retinal degenerations (RDs), in the advent of clinical trials testing treatment options.

Conclusions: LRAT-associated phenotypes in this cohort were variable and unusual, but generally milder than those seen in RPE65-associated disease, and may be particularly amenable to treatment. The window of therapeutic opportunity can be extended in patients with a mild phenotype.

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