treatment of rp

/Tag: treatment of rp

To Treat an Inherited Retinal Disease….

In simple terms, genes are like recipes for making proteins. All the cells in our bodies “read” genetic information so they can make the critical proteins necessary to stay healthy and function properly. If there is a mistake in a gene — that is, a misspelling — a protein might not be made correctly and [...]

By |۱۳۹۶/۸/۹ ،۰۶:۴۷:۴۳ +۰۰:۰۰آبان ۹ام, ۱۳۹۶|English, دسته‌بندی نشده|0 Comments

The first gene therapy for the treatment of rp

Phase 1 studies have shown potential benefit of gene replacement in RPE65-mediated inherited retinal dystrophy. This phase 3 study assessed the efficacy and safety of voretigene neparvovec in participants whose inherited retinal dystrophy would otherwise progress to complete blindness. Methods In this open-label, randomised, controlled phase 3 trial done at two sites in the USA, [...]

By |۱۳۹۶/۷/۲۲ ،۰۷:۴۲:۵۶ +۰۰:۰۰مهر ۲۲ام, ۱۳۹۶|English, دسته‌بندی نشده|0 Comments