… Choroideremia is an X-linked retinal degeneration resulting … from clinical trials in
choroideremia, review endpoints, and … be used as endpoints must be considered for …

… Choroideremia is an X-linked retinal degeneration resulting … from clinical trials in
choroideremia, review endpoints, and … be used as endpoints must be considered for …

… endpoint despite demonstrating some statistically significant improvements, such as Biogen
phase 3 STAR trial in choroideremia… IRD trials require novel, clinically meaningful endpoints …

… promising treatment options for choroideremia and other IRDs… Choroideremia is a good
candidate for gene augmentation … the primary endpoints and key secondary endpoints after 12 …

… treat choroideremia, 3 a disease that is characterized by atrophy of the choriocapillaris and
retinal pigment epithelium and involves vision loss that leads to blindness. Choroideremia is …

Inherited retinal dystrophies (IRDs) encompass a group of genetically diverse disorders, each
uniquely influencing distinct retinal cell pathways and retinal areas. IRDs currently affect …

… The most prevalent example, choroideremia (CHM), will be discussed in this review. …
Choroideremia is caused by a mutation in the CHM gene, which encodes the Rab escort protein 1 (…

… term (60-week endpoint) (Figure 4). … endpoint, vector copies in the brain were substantially
higher in the Gm2a −/− mice treated at 6-weeks of age than they were at the 20-week endpoint…

… Although some authors suggested an age-dependent penetrance for choroideremia [17],
in our patient, an early onset of the phenotype has been documented. As reported for females …

… Choroideremia is an X-linked IRD caused by mutations in the CHM gene. Choroideremia …
have indicated a failure to meet primary endpoints, as improvements from baseline best …