RETINAL DYSTROPHY

Clinical trials for RETINAL DYSTROPHY explained in plain language.

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Trials to join now! 2 Not yet finished but already full! 1 Completed 1

Custom-Made genetic drug targets rare blindness in single patient

Disease control Ongoing

This study tests a custom-made genetic drug (antisense oligonucleotide) designed for one person with retinal dystrophy caused by a specific PRPH2 gene mutation. The goal is to see if the drug is safe and can slow or stop vision loss. Only one participant will receive the treatmen…

Matched conditions: RETINAL DYSTROPHY

Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control

Last updated May 12, 2026 13:41 UTC

RETINAL DYSTROPHY

Get alerted when new RETINAL DYSTROPHY trials appear

Custom-Made genetic drug targets rare blindness in single patient