Changing a Treatment Landscape by De-Risking Drug Development

Spinocerebellar ataxia is a group of inherited, heterogeneous neurodegenerative diseases affecting coordination, speech, and vision. There are currently no FDA approved therapies for the more than 50 known types of SCA​, but there is a growing pipeline of therapeutic candidates. We spoke to Andrew Rosen, CEO of the National Ataxia Foundation, about the challenges of developing therapies for spinocerebellar ataxia, the critical role of patient-led organizations in early-stage research and advocacy, and the recent surge of therapeutic activity targeting these neurodegenerative conditions.