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About Us

Our Leadership

Collaborations

Compliance & Ethics

Corporate Giving

Our Focus

Cerebral Adrenoleukodystrophy

Sickle Cell Disease

Transfusion Dependant β-Thalassemia

Our Science

What is Gene Therapy?

Our Pipeline

Clinical Trials 

Our Products

Patients & Advocacy

Patient Advocacy

Patient Resources

Access Policy 

Contact Us

Investors & Media

Careers

Stories

 

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