The photoreceptors of the retina are afflicted by diseases that still often lack satisfactory treatment options. Although suitable drugs might be available in some cases, the delivery of these compounds into the eye and across the blood–retinal barrier remains a significant challenge for therapy development. Here, we review the routes of drug administration to the retina and highlight different options for drug delivery to the photoreceptor cells.
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Hi, I'd like to have more information on HJMD and its treatments. Has the Ataulauren produced any positive results?
Unfortunately, there is no explicit efficacy study in HJMD with any kind of read-through drug.
In the Moorfields Eye Hospital London is Mrs. Mariya Moosajee and in Mainz Germany the group Wolfrum have already published a lot here.
Of course, there is a lack of money - what a pity, because with a functioning substance, one could effectively treat a great many genetic diseases with it.
Thanks for the reply. In Italy, I followed a case of HJMD through the Gaslini hospital in Genoa where a sample of the patient's cells was treated with Ataulauren at the Catanzaro laboratory. Unfortunately, there have been no positive feedbacks to date. Do you have any tips to limit macular degeneration?
I have sent you an email with further information and questions.
I hope the email address is correct and has arrived.