Articles by tag: rare-disease

Gene therapy OPGx-LCA5 accepted into FDA RDEP program

Learn how Opus Genetics' gene therapy OPGx-LCA5 received RDEP status from the FDA, accelerating the development of treatment for ultra-rare LCA5 blindness in children. Detailed analysis and prospects.

Zilganersen improved walking speed in Alexander disease

Antisense therapy with zilganersen shows first success in Alexander disease. Learn about phase 1-3 studies where the drug improved walking speed and stabilized patient condition.