References in periodicals archive ?
The trial will be structured as a monocentric, open-label, dose-escalation, Phase I/II study and will investigate the compound's impact on 12 patients aged 18 years or older, who are suffering from a retinitis pigmentosa caused by mutations in the PDE6B gene, with no other syndromic manifestations.
"We are pleased to initiate this Phase I/II trial to confirm the safety of HORA PDE6B, administered via unilateral subretinal injection, for the treatment of this form of retinitis pigmentosa."
Morgan et al., "Genotype-phenotype correlation of mouse Pde6b mutations," Investigative Ophthalmology and Visual Science, vol.