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Areas of Focus

MPS I patient

RARE DISEASES

35+

years pioneering research for patients living with rare genetic conditions

Multiple sclerosis patient

MULTIPLE SCLEROSIS

2.3

million people living with multiple sclerosis worldwide

2

therapies approved for immune-mediated diseases with several more in clinical trials

Atopic Dermatitis patient

IMMUNOLOGY

6

late-stage research programs focused on discovering new options for cancer patients

Prostate cancer patient

ONCOLOGY

Press Release

FABRY DISEASE

Living with Fabry Disease Then and Now

2018-08-08

Image of Evis, Multiple Sclerosis Researcher

MULTIPLE SCLEROSIS

A Conversation with Multiple Sclerosis Researcher, Evis Havari

2018-05-30

Image of Paula, MPS I disease patient

MPS I DISEASE

Paula’s MPS I Story

2018-05-15

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Research Pipeline

EXPLORE R&D HIGHLIGHTS

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Careers

DISCOVER OPPORTUNITIES

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Patient Access to Treatment

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