1 Corporate Overview Delivering Life-Changing Therapies to People Living with Rare Diseases October 2019 Forward-Looking Statements This presentation contains forward-looking statements, including statements about our prospects, products, growth projections, competitive position, potential regulatory filings and agency actions, and the anticipated development, timing, data readouts and therapeutic scope of programs in our clinical pipeline. These forwardlooking statements may be accompanied by such words as ...

1 Corporate Overview Delivering Life-Changing Therapies to People Living with Rare Diseases March 2019 Forward-Looking Statements This presentation contains forward-looking statements, including statements about our prospects, products, growth projections, competitive position, potential regulatory filings and agency actions, and the anticipated development, timing, data readouts and therapeutic scope of programs in our clinical pipeline. These forward-looking statements may be accompanied by such words as ...

1 Corporate Overview Delivering Life-Changing Therapies to People Living with Rare Diseases January 2019 Forward-Looking Statements This presentation contains forward-looking statements, including statements about our prospects, products, growth projections, competitive position, potential regulatory filings and agency actions, and the anticipated development, timing, data readouts and therapeutic scope of programs in our clinical pipeline. These forward-looking statements may be accompanied by such words a ...