Upcoming Treatments

Many exciting new treatments are being developed for inherited retinal dystrophies, including:

Ciliary Neurotrophic Factor (CNTF) and Encapsulated Cell Technology (ECT)

CNTF is a protective factor that is being developed as a treatment for inherited retinal diseases.  It involves a surgery where a small implant is placed inside the eye, which then secretes CNTF. It has been shown to slow disease progress significantly, and in some cases even recover some vision!

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Argus II Retinal Prosthesis (microchip)

The Argus II Retinal Prosthesis is a microchip that is surgically implanted onto the retina. The patient can then wear a pair of glasses with an attached camera which transmits visual information to the retina! It allows patients with extremely poor vision to recover some visual acuity and function. The Argus II has recently been approved by the FDA.

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9-cis retinyl acetate

9-cis retinyl acetate has recently been investigated in a phase 1b trial as an oral drug therapy for patients with certain mutations causing Leber’s Congenital Amaurosis. These patients were shown to have increased visual function after a course of 9-cis retinylacetate
Gene replacement therapy

Gene therapy is being investigated for a number of retinal diseases. Gene therapy involves replacement of the specific mutated/dysfunctional gene, and therefore allows reversal of the disease process. Recently, successful gene replacement therapy was performed in patients with Choroideremia!

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Stem Cell Therapy

Although it is further away than other treatments, stem cell therapy is being investigated as a potential cure for many inherited retinal dystrophies, and animal models have shown promising results!