Characterized by progressive deafness, blindness and variable balance disorder, Usher syndrome type III (USH3) is caused by destabilizing mutations in the gene encoding the clarin-1 protein (CLRN1). This webinar details a successful new strategy to identify small molecule therapies to mitigate the hearing loss associated with these mutations, highlighting the promise of this discovery approach in the quest for drugs to treat other protein-destabilizing monogenic disorders.