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Leber Congenital Amaurosis 10 research hub
2 indexed studies · 2 currently recruiting
- recruitingPHASE3
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
This study is testing a new medicine called sepofarsen for a rare eye condition called Leber Congenital Amaurosis Type 10 (LCA10). It aims to see if sepofarsen can improve vision safely in people with this condition.
United States · Belgium · Brazil - recruiting
Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center
This study looks back at records of over 1,200 patients in France who had a rare blood disorder called iTTP. It aims to understand how things like heart problems and specific treatments affect their long-term health and survival. It hopes to improve care for people with iTTP.
France