Patients & families
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Working together to make curative treatments a possibility.
We are devoted to creating better lives for people living with chronic debilitating disease. We work with families and patient organisations to ensure our programmes deliver the best treatments and resources for gene therapy and genetic disease.
Link to: Gene therapy
Gene therapy
We are transforming our innovative gene technology into potentially life-changing treatments.
Link to: Lysosomal Storage Disorders
Lysosomal Storage Disorders
We are working to bring a potential first- and best-in-class gene therapy to patients with Gaucher disease Type 1.
Link to: Patient organisations
Patient organisations
The following organisations offer resources and information to help people affected by Gaucher disease