This website is an educational service brought to you by BioMarin and is intended for residents of Europe

TURNING COMPLEXITY
INTO CLARITY

AN UNDERSTANDABLE RESOURCE ON GENE THERAPY RESEARCH

About HaemDifferently

HaemDifferently aims to open an honest conversation around the topic of gene therapy research. What you can expect from this site, or any of the other HaemDifferently resources, is accurate and straightforward information.

Providing information that can help make gene therapy more familiar and approachable is important to us, but we know we can’t do this alone. We are incredibly grateful to the gene therapy investigators, haemophilia community, advocacy groups and linguistic specialists who have helped inform and shape this educational platform. Together, we believe we created something that will help many people, across all walks of life, along their journey to better understanding the science behind gene therapy research.

How is gene therapy designed to work?

It’s not magic, it’s science in progress

GENE THERAPY RESEARCH

WHAT ARE YOU CURIOUS ABOUT?

WHAT IS GENE THERAPY?

WHAT IS A GENE?

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WHAT CAUSES GENETIC CONDITIONS?

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WHAT CAUSES GENETIC CONDITIONS?

What is gene therapy?

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How is gene therapy designed to work?

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WHAT ARE THE GOALS OF GENE TRANSFER THERAPY RESEARCH?

WHAT ARE THE GOALS OF GENE THERAPY RESEARCH?

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WHAT ARE THE POSSIBLE RISKS OF GENE THERAPY RESEARCH?

WHAT are the possible RISKS OF GENE THERAPY?

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