Therapeutic regimen and methods for treating or ameliorating visual disorders associated with an endogenous retinoid deficiency

摘要

Disclosed herein are therapeutic regimens for treating or ameliorating a visual disorder associate with an endogenous retinoid deficiency in a subject by administering a therapeutically effective amount of a synthetic retinal derivative or a pharmaceutically acceptable composition comprising a synthetic retinal derivative according to the therapeutic regimen which leads to local recovery of visual functions such as visual fields, visual acuity and retinal sensitivity, among others.

主权项

1. A method for treating a human patient suffering from the loss or impairment of vision caused by Leber Congenital Amaurosis (LCA), wherein said patient is deficient in endogenous 11-cis-retinal disorder, comprising the steps of:
a) administering to the human patient an initial therapeutically effective dose of a synthetic retinal derivative selected from the group consisting of 9-cis-retinyl acetate, 9-cis-retinyl formate, 9-cis-retinyl succinate, 9-cis-retinyl citrate, 9-cis-retinyl ketoglutarate, 9-cis-retinyl fumarate, 9-cis-retinyl malate, 9-cis-retinyl oxaloacetate, 9-cis-retinyl propionate, 9-cis-retinyl butyrate, 9-cis-retinyl valerate, 9-cis-retinyl hexanoate, 9-cis-retinyl heptanoate, 9-cis-retinyl octanoate, 9-cis-retinyl nonanoate, 9-cis-retinyl decanoate, 9-cis-retinyl undecanoate, 9-cis-retinyl dodecanoate, 9-cis-retinyl oxalate, 9-cis-retinyl malonate, 9-cis-retinyl glutarate, 9-cis-retinyl adipate, 9-cis-retinyl pimelate, 9-cis-retinyl suberate, 9-cis-retinyl azelate, and 9-cis-retinyl sebacate, b) refraining from administering to the human patient an additional therapeutically effective dose of the synthetic retinal derivative for a resting interval, wherein the resting interval is a time period between 1 month and 14 months, c) administering to the human patient the additional therapeutically effective dose of the synthetic retinal derivative.