No is a word they’ve
heard too often.

Most patients with severe and life-threatening rare diseases live with no effective treatment options and no hope. We develop and deliver treatments that will dramatically transform their lives.

No is a word they’ve
heard too often.

Most patients with severe and life-threatening rare diseases live with no effective treatment options and no hope. We develop and deliver treatments that will dramatically transform their lives.

No is a word they’ve
heard too often.

Most patients with severe and life-threatening rare diseases live with no effective treatment options and no hope. We develop and deliver treatments that will dramatically transform their lives.

We work relentlessly...

Our entire global organization is focused on bringing breakthrough therapies to patients in need. Today, we are serving patients in nearly 50 countries and advancing the most robust pipeline of innovative therapies in our history.

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Making dramatic discoveries for patients with severe and life-threatening diseases.

Our development pipeline is focused on highly innovative therapies with the potential to transform the lives of patients with devastating diseases who are often overlooked because of the rarity of their disorders.

Making dramatic discoveries for patients with severe and life-threatening diseases

Our development pipeline is focused on highly innovative therapies with the potential to transform the lives of patients with devastating diseases who are often overlooked because of the rarity of their disorders.

Because if they ask whether there’s a treatment...

Our global team is driven to provide patients with hope.

Because if they ask whether there’s a treatment...

Our global team is driven to provide patients with hope.

Because if they ask whether there’s a treatment...

Our global team is driven to provide patients with hope.

We need to tell them yes.

Every day we challenge ourselves to reach the ultimate goal of telling patients, “Yes,” there is a treatment. “Yes,” you will have access to it. And “Yes,” we’ll continue to serve patients by introducing new life-transforming treatments worldwide.

We need to tell them yes.

Every day we challenge ourselves to reach the ultimate goal of telling patients, “Yes,” there is a treatment. “Yes,” you will have access to it. And “Yes,” we’ll continue to serve patients by introducing new life-transforming treatments worldwide.

We need to tell them yes.

Every day we challenge ourselves to reach the ultimate goal of telling patients, “Yes,” there is a treatment. “Yes,” you will have access to it. And “Yes,” we’ll continue to serve patients by introducing new life-transforming treatments worldwide.