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First small molecule targeted therapy to mitigate hearing loss in Usher syndrome type 3

Usher syndrome (USH) is characterized by hearing loss or deafness at birth and progressive vision loss, and is the most common cause of inherited dual sensory deficit.

No treatment is currently available to stop or slow the progression of vision or hearing loss in USH3, one of three clinical classifications for USH that are further divided into subtypes and all associated with different genes.