Badanie nad bezpieczeństwem i tolerancją SAR421869 u pacjentów z zespołem Ushera typu 1B

Usher syndrome is a rare genetic disorder that affects both hearing and vision. This study focuses on a specific type of this condition, known as Usher syndrome Type 1B. The purpose of the study is to evaluate the long-term safety and tolerability of a treatment called SAR421869. This treatment involves a lentiviral vector, which is a type of virus modified to carry a specific human gene, in this case, the MYO7A gene. The MYO7A gene is important for normal hearing and vision, and its alteration is linked to Usher syndrome Type 1B.

The treatment SAR421869 is administered as a suspension for injection. The study aims to observe how patients respond to this treatment over a long period. Researchers will monitor any side effects and assess whether the treatment can slow down the progression of vision loss associated with the syndrome. The study is open-label, meaning both the researchers and participants know what treatment is being given.

Participants in the study have previously received a subretinal injection of SAR421869, which means the treatment was injected into a specific area of the eye. The study will track any adverse events and look for significant changes in eye health. The goal is to determine if the treatment is safe and if it can help delay the degeneration of the retina, which is the part of the eye that is crucial for vision. The study is expected to continue for several years to gather comprehensive data on the treatment's effects.